Upcoming webinar: Patient and clinician involvement in the EU HTA regulation

Myeloma Patients Europe (MPE) and ALAN invite you to register to the webinar “Patient and clinician involvement in the EU Health Technology Assessment (HTA) regulation”

Date and time: Wednesday 26th June 2024, 15:00 – 16:30 CEST.

Link to register:

https://mpeurope-org.zoom.us/webinar/register/WN_bJjmcrVuR46CEtwKxRmU6g#/registration

What can you expect ?

The webinar will be structured as a multistakeholder panel discussion on the EU HTA regulation and its impact concerning patient and clinician involvement.

Speakers

• Anke-Peggy Holtorf, moderator, Steering Committee member and project coordinator for the Patient and Citizen Involvement in HTA Interest Group, Health Technology Assessment international (HTAi)

• François Houÿez, Treatment Information and Access Director / Health Policy Advisor, European Organisation for Rare Diseases (EURORDIS)

• Mihai Rotaru, Director Market Access, European Federation of Pharmaceutical Industries and Associations (EFPIA) • Mirjana Huic, HTA independent specialist in HTA/EBM Centre, Croatia

• Robin Doeswijk, Head of European Affairs, European Hematology Association (EHA)

• Representative of a payer organisation (TBC).

More information:

Registration to this event is free of charge. Feel free to forward the registration link to colleagues and other patient advocates who might be interested in attending the webinar. Should you be unable to attend the webinar, please feel free to send your questions in advance to info@mpeurope.org and we will make sure they reach our speaker. A recording of the webinar will be available shortly after the session. Keep an eye out for our social media profiles!

We look forward to your participation.

ALAN at EHA Congress: Empowering advocates for acute leukemia awareness!

We’re thrilled to be attending the European Hematology Association (EHA) Congress in Madrid from June 13-16. Get ready for a series of engaging activities that advance awareness and knowledge about acute leukemia, as we delve into the latest research from EHA.

Following EHA, don’t miss the opportunity to join our exclusive webinar featuring expert speakers discussing the latest topics in acute leukemia and unraveling key insights from the congress.

Furthermore, stay tuned for comprehensive reporting and infographics highlighting key takeaways and developments presented at EHA.

More information on each of these initiatives can be found below:

WEBINAR – Unravelling EHA’s key topics: Patient and HCP perspectives

Date: Tuesday July 2nd at 12:30 – 1:30PM CEST

Registration link: https://us06web.zoom.us/webinar/register/WN_jXuX3vpHQOmkW3wd2E_-xw

Join us for an exclusive webinar hosted by ALAN following the EHA Congress! Dive into key topics presented at EHA alongside. In this dynamic 60-minute session, we’ll explore the significance of these topics for patients, with ample opportunities for live Q&A.

REPORTS – EHA Congress presentation summaries

Discover the latest insights and developments shared at the EHA Congress. Here, you’ll find concise summaries of presentations exploring various aspects of acute leukemia, unraveling their significance to the patient community.

Visit our website during the week of June 24 to catch these new updates.

INFOGRAPHIC – Welcome to our infographic gallery

Download your copy and discover the core insights from EHA Congress, condensed into a dynamic infographic.

Stay tuned for the release of our infographic, coming the week commencing July 01.

Webinar: Awareness of BPDCN

ALAN organized a free webinar focusing on BPDCN, blastic plasmacytoid dendritic cell neoplasm, a rare type of acute leukemia that most commonly manifests as cutaneous lesions with or without bone marrow involvement and leukemic dissemination.

For most of us, BPDCN is not known. Join us for this webinar to learn about it !

Presented by Dr Cristina Papayannidis, MD, PhD

IRCCS Azienda Ospedaliero-Universitaria di Bologna – Istituto di Ematologia “Seràgnoli” , Bologna, Italy

Adjunct Professor of Hematology – Alma Mater Studiorum, University of Bologna

Presentation

BPDCN_Dr Cristina Papayannidis

Recording

Webinar: The new EU HTA regulation and implications for European and national patient organisations and patients

Myeloma Patients Europe (MPE) and ALAN held a webinar on 22nd May 2024 on “The new EU Health Technology Assessment (HTA) regulation and implications for European and national patient organisations and patients”

What can you expect ?

The webinar provided an overview of the main provisions of the new EU HTA regulation with a focus on how these provisions will involve and impact patients, followed by a Q&A session with participants.

Speaker

The guest speaker was Béla Dajka, policy officer at the Directorate-General for Health and Food Safety in the European Commission. He is part of the team working on the implementation of the EU HTA regulation. Prior to this role, he oversaw EU support to national health systems for several EU countries. Béla studied journalism, media, management and health economics at universities in Russia, UK and USA. He worked at the BBC from 1998 to 2007 and joined the European Commission in 2008.

Recording:

Q1 2024: Additional information and data in acute leukemia

ALL

Autolus Therapeutics announces acceptance of Biologics License Application (BLA) for obecabtagene autoleucel (obe-cel) as a potential treatment for relapsed/refractory Adult B-cell ALL.

Obe-cel is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies. Obe-cel is designed with a fast target binding off-rate to minimize excessive activation of the programmed T cells. Clinical trials of obe-cel have demonstrated that this “fast off-rate” profile reduces toxicity and T cell exhaustion, resulting in improved persistence and leading to high levels of durable remissions in r/r Adult ALL patients. The results of the FELIX trial, a pivotal trial for adult ALL, are being prepared for regulatory submissions with the FDA and EMA. Autolus is conducting a Phase 1b study in paediatric patients with ALL and aggressive B-NHL and iIn collaboration with UCL, obe-cel is currently being evaluated in a Phase 1 clinical trials for B-NHL.

The BLA submission is based on data from the Pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL. Autolus plans to submit a Marketing Authorization Application for obe-cel in relapsed/refractory ALL to the European Medicines Agency (EMA) in the first half of 2024.

Press release –> read here.

Takeda Announces U.S. FDA Approval of Supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) in Adult Patients with Newly Diagnosed Ph+ ALL. Press release –> read here

A phase 1/2 study of mini-hyper-CVD plus venetoclax in patients with R/R ALL:

Preclinical studies suggest that Bcl-2 inhibition with venetoclax has antileukemic activity in ALL and may synergize with conventional chemotherapy. In this trial, patients received the mini-hyper-CVD regimen (dose-attenuated hyperfractionated cyclophosphamide, vincristine, and dexamethasone alternating with methotrexate and cytarabine) in combination with venetoclax (200 mg or 400 mg daily) on days 1 to 14 in cycle 1 and on days 1 to 7 in consolidation cycles. The recommended phase 2 dose of venetoclax in the combination regimen was 400 mg daily. The composite complete remission (CR) and CR with incomplete hematologic recovery (CRi) rate was 57% (CR, 43%; CRi, 14%), and 45% of responders achieved measurable residual disease negativity by multiparameter flow cytometry. Four patients proceeded to allo-SCT. The median duration of response was 6.3 months. The median overall survival was 7.1 months, and the 1-year overall survival rate was 29%. The most common grade ≥3 nonhematologic adverse events were infection in 17 patients (77%) and febrile neutropenia in 4 patients (18%). Overall, the combination of mini-hyper-CVD plus venetoclax was active in heavily pretreated R/R ALL. Further development of venetoclax-based combinations in ALL is warranted. (Ref: https://doi.org/10.1182/bloodadvances.2023012231)

T-PLL

Limited efficacy for ibrutinib and venetoclax in T-prolymphocytic leukemia: results from a phase 2 international study –> read here

AML

Venetoclax–based low intensity therapy in molecular failure of NPM1-mutated AML

Molecular failure in NPM1-mutated AML inevitably progresses to frank relapse if untreated. Recently published small case series show that venetoclax combined with low-dose cytarabine or azacitidine can reduce or eliminate measurable residual disease (MRD). Here, we report on an international multicenter cohort of 79 patients treated for molecular failure with venetoclax combinations and report an overall molecular response (≥1-log reduction in MRD) in 66 patients (84%) and MRD negativity in 56 (71%). Eighteen of 79 patients (23%) required hospitalization, and no deaths were reported during treatment. Forty-one patients were bridged to allogeneic transplant with no further therapy, and 25 of 41 were MRD negative assessed by reverse transcription quantitative polymerase chain reaction before transplant. Overall survival (OS) for the whole cohort at 2 years was 67%, event-free survival (EFS) was 45%, and in responding patients, there was no difference in survival in those who received a transplant using time-dependent analysis. Presence of FLT3-ITD mutation was associated with a lower response rate (64 vs 91%; P < .01), worse OS (hazard ratio [HR], 2.50; 95% confidence interval [CI], 1.06-5.86; P = .036), and EFS (HR, 1.87; 95% CI, 1.06-3.28; P = .03). Eighteen of 35 patients who did not undergo transplant became MRD negative and stopped treatment after a median of 10 months, with 2-year molecular relapse free survival of 62% from the end of treatment.

Venetoclax–based low intensive chemotherapy is a potentially effective treatment for molecular relapse in NPM1-mutated AML, either as a bridge to transplant or as definitive therapy. (Ref: https://doi.org/10.1182/bloodadvances.2023011106)

Molecular MRD is strongly prognostic in patients with NPM1-mutated AML receiving venetoclax-based nonintensive therapy

Assessment of measurable residual disease (MRD) is strongly prognostic in patients with NPM1-mutated AML treated with intensive chemotherapy; however, there are no data regarding its utility in venetoclax-based nonintensive therapy, despite high efficacy in this genotype.

A study analyzed the prognostic impact of NPM1 MRD in an international real-world cohort of 76 previously untreated patients with NPM1-mutated AML who achieved complete remission (CR)/CR with incomplete hematological recovery following treatment with venetoclax and hypomethylating agents (HMAs) or low-dose cytarabine (LDAC). A total of 44 patients (58%) achieved bone marrow (BM) MRD negativity, and a further 14 (18%) achieved a reduction of ≥4 log10 from baseline as their best response, with no difference between HMAs and LDAC. The cumulative rates of BM MRD negativity by the end of cycles 2, 4, and 6 were 25%, 47%, and 50%, respectively. Patients achieving BM MRD negativity by the end of cycle 4 had 2-year overall of 84% compared with 46% if MRD was positive. On multivariable analyses, MRD negativity was the strongest prognostic factor. A total of 22 patients electively stopped therapy in BM MRD-negative remission after a median of 8 cycles, with 2-year treatment-free remission of 88%.

In patients with NPM1-mutated AML attaining remission with venetoclax combination therapies, NPM1 MRD provides valuable prognostic information. (ref: https://doi.org/10.1182/blood.2023021579)

EHA Adolescent and Young Adult Patient Advocates

Many blood diseases occur in later phases of life, but in each of them, there are also many young patients who need to deal with these conditions, symptoms and therapies. These patients have specific needs in terms of treatment and research, being able to perform at school or work, fertility, survivorship as well as sexual and mental health.
In the European Patient Advocacy Institute they have been running an exciting training programme of the European Hematology Association (EHA) for adolescent and young adults (AYA) patient advocates. This weekend, they had the first meeting with 15 young patient advocates from CML Advocates Network, Global MPN Advocates Network, Acute Leukemia Advocates Network, CLL Advocates Network, Myeloma Patients Europe (MPE), ITP Support Association, European Sickle Cell Federation (ESCF) and the PNH Global Alliance.
The objective is to strengthen patient advocacy groups to better address the specific needs of young patients and survivors affected by different hematological diseases.The training aims to equip the trainees with the essential knowledge and capabilities needed for impactful advocacy and foster the collaboration with the EHA AYA Taskforce. The training programme is designed as a hybrid model of in-person and virtual sessions.
During the meeting they jointly identified key unmet needs of AYA patients in blood diseases, and brainstormed about potential advocacy actions that could address those. They also held a training sessions about patient advocacy and using evidence, and had young hematologists presenting about malignant and non-malignant blood diseases.
We will see them during the EHA congress in June and we look forward to more discussions !

smartCARE project

The EU-funded smartCARE project is creating a mobile application to help cancer survivors improve their health and wellbeing. A prototype is currently under development and will be shaped by feedback from cancer patients, survivors and caregivers.

The smartCARE project wants to hear from cancer survivors and caregivers across Europe!

Please share the survey with your community. The responses will advise developers to build a user-focused app: https://bit.ly/3t1hIdU

All respondents will be eligible for free virtual registration to the 2024 European Cancer Summit.

#EUsmartCARE #CancerCare #Oncology #smartCAREsurvey

Q4 2023: Additional information and data in acute leukemia

APL

Impact of the ICAL on the treatment of acute leukemia; The International Consortium on APL (IC-APL) is an initiative of the International Members Committee of the American Society of Hematology, created in the spirit of international clinical and laboratory collaboration with the aim of reducing the difference in the outcomes of patients with APL treated in developed and developing countries. It congregates leaders of well-established cooperative groups in Europe and North America, and hematologists in Brazil, Chile, Peru, Uruguay, and Paraguay. APL was initially selected as a model disease to test the impact of networking on outcomes because it is a highly curable disease if early diagnosis and specific treatment are promptly established. Full article: https://doi.org/10.1182/bloodadvances.2017002147

ALL

Nelarabine: when and how to use in the treatment of T-cell ALL

T-cell acute lymphoblastic leukemia or lymphoblastic lymphoma (T-ALL/LBL) is a rare hematologic malignancy most commonly affecting adolescent and young adult males. Outcomes are dismal for patients who relapse, thus, improvement in treatment is needed. Nelarabine, a prodrug of the deoxyguanosine analog 9-β-arabinofuranosylguanine, is uniquely toxic to T lymphoblasts, compared with B lymphoblasts and normal lymphocytes, and has been developed for the treatment of T-ALL/LBL. Based on phase 1 and 2 trials in children and adults, single-agent nelarabine is approved for treatment of patients with relapsed or refractory T-ALL/LBL, with the major adverse effect being central and peripheral neurotoxicity. Since its approval in 2005, nelarabine has been studied in combination with other chemotherapy agents for relapsed disease and is also being studied as a component of initial treatment in pediatric and adult patients.

Nelarabine represents a valuable agent for the treatment of T-ALL. The strongest evidence supports the use of nelarabine for the treatment of early relapse, with retrospective data suggesting improved chance of response when used in combination with other chemotherapy such as CTX and etoposide. Durable clinical benefit requires consolidation with allo-HSCT. The role of nelarabine in the initial treatment of T-ALL is less established, although supported for treatment of young patients with HR T-ALL in combination with the COG 0434 backbone with benefit related to reduction in CNS relapse. The benefit of nelarabine in other contexts including in combination with other frontline adult and pediatric regimens, and for treatment of persistent MRD, is not established. For those treated with nelarabine, neurotoxicity appears to be a modest and manageable risk in appropriately selected patients but vigilance is required. (Ref: https://doi.org/10.1182/bloodadvances.2023010303)

Short_NGS vs BCR-Abl PCR_AmJHematol 2023

The takeaways from the data are:

  • clonoSEQ is more prognostic in Ph+ ALL than traditional BCR-Abl RT-PCR
  • The differing prognostic values of these two assays for these patients are consistent with other reports that the lymphoid component (as measured by clonoSEQ) is distinct from, and more aggressive than, the myeloid component (as measured by BCR-Abl RT-PCT).
AML

Midostaurin plus daunorubicin or idarubicin for young and older adults with FLT3-mutated AML: RATIFY phase 3b trial:

In this study, midostaurin in combination with intensive chemotherapy provided high response rates, irrespective of patient age, induction regimen (“7+3” or “5+2”), or the type of anthracycline used (daunorubicin or idarubicin) during the induction therapy. Overall, 80.7% of patients achieved CR + CRi and 65.3% of patients achieved CR, with similar rates observed in patient subgroup analyses based on age, induction regimen, induction drug, and gender. These response rates support the results observed in the RATIFY study, in which 59% of patients who received midostaurin achieved CR.21 The results from this study also align with those from a phase 2 study (the German-Austrian AMLSG trial) of midostaurin in combination with standard induction and consolidation chemotherapy, followed by SCT, which allowed for enrollment of adult patients aged up to 70 years; similar CR + CRi rates were observed after induction in younger and older patients, with a manageable safety profile.24,25 Furthermore, the multivariate analysis from the AMLSG trial showed significant beneficial effect of midostaurin on EFS and OS in both younger and older patients (61-70 years). Full article: https://doi.org/10.1182/bloodadvances.2023009847

Phase 1 study of vibecotamab identifies an optimized dose for treatment of relapsed/refractory AML: 

 Identification of a recommended phase 2 vibecotamab dose comprised 3 step-up doses (Week 1), which were noted to reduce cytokine response syndrome (CRS), followed by weekly dosing (1.7 μg/kg, Cohort -1D). In 16 of 120 patients, at least 1 treatment-emergent adverse event was classified as a dose-limiting toxicity. CRS, the most common adverse event (59.2%), managed with premedication, were mostly ≤grade 2. A secondary objective was assessment of efficacy in patients with CD123-expressing leukemias. A total of 10 of 111 (9.0%) efficacy-evaluable patients with AML achieved an overall response of morphologic leukemia-free state or better with an overall objective response rate (ORR) of 9.0%. Response was only observed in patients receiving a target dose of 0.75 μg/kg or higher (n = 87) in which the efficacy-evaluable ORR was 11.5%. Response was associated with lower baseline blast counts in blood and bone marrow (<25%) suggesting potential benefit. (Ref: https://doi.org/10.1182/bloodadvances.2023010956)

Sorafenib plus intensive chemotherapy in newly diagnosed FLT3-ITD AML: a randomized, placebo-controlled study by the ALLG

Loo et al report the results of a phase 2, placebo-controlled study of adding sorafenib to intensive induction chemotherapy for patients with FLT3-ITD AML. The authors report that event-free survival and overall survival were not improved by sorafenib in this setting. However, the study suggests that sorafenib may improve survival in those patients undergoing transplant in first remission; a larger study would be required to confirm this. (Ref; https://doi.org/10.1182/blood.2023020301).

Cigarette smoke exposure accelerates AML progression in FLT3-ITD models

Past work reports smokers with AML have worse survival outcomes than nonsmokers; however, to the best of our knowledge, this is the first study to model cigarette smoke exposure in FLT3-mutant AML–bearing mice to examine potential molecular mediators of leukemia progression and chemotherapy resistance. SE accelerated disease progression in 3 FLT3-ITD AML mouse models. SC upon leukemia engraftment slowed acceleration, providing the first evidence that smoking and cessation deliver “go” and “no-go” signals to FLT3-ITD AML cells. Because many patients with cancer continue smoking after their diagnosis, these data from xenograft models provide evidence for cessation recommendations, but it will require further validation in primary AML samples. Additionally, mass cytometry revealed that SE increased protein expression of MCL-1, DNMT3B, and RUNX1. MCL-1 inhibitors are currently being investigated for AML treatment, especially in association with resistance to venetoclax,and RUNX1 mutations occur in 10% of patients with AML and are associated with inferior prognosis. Cumulatively, our data provide novel insights into previously undescribed molecular regulators of aggressive disease seen in patients with AML with histories of smoking. (Ref: https://doi.org/10.1182/bloodadvances.2023010111)

Aspacytarabine for the treatment of patients with AML unfit for intensive chemotherapy: a phase 2 study:

High-dose cytarabine is associated with gastrointestinal and cerebellar toxicity, precluding its use for older or unfit patients with acute myeloid leukemia (AML). Aspacytarabine, an inactive prodrug of cytarabine, was evaluated as monotherapy in a phase 2b study of patients unfit for intensive chemotherapy (NCT03435848). Sixty-five patients with AML were treated with aspacytarabine 4.5 g/m2 per day (equimolar to 3 g/m2 per day cytarabine) for 6 doses per treatment. The median age was 75 years; 60.6% of patients had de novo AML, 28.8% had AML secondary to myelodysplastic syndrome, and 10.6% had therapy-related AML. Overall, 36.9% achieved complete remission (CR) with full count recovery. CR rates in patients with secondary AML, patients with prior treatment with hypomethylating agents, and patients with TP53 mutation were 26.7%, 25%, and 36%, respectively. Median overall survival was 9 months (range, 6-15.9) and was not reached among responders. Hematologic recovery was observed in all responding patients by day 26 without prolonged cytopenias. Adverse events typically precluding the use of high-dose cytarabine in older or unfit patients were not observed.

These data suggest that aspacytarabine may be an effective regimen with a reduction in the attendant toxicities associated with high-dose cytarabine, an important consideration when treating AML and other hematologic disorders that use high-dose cytarabine. (ref: https://doi.org/10.1182/bloodadvances.2023010943)

Vitamin C and D supplementation in AML

  • Vitamin C/D treatment was associated with less complications during chemotherapy and restores the vitamin D level before allogeneic hematopoietic cell transplantation in patients with AML.

  • Vitamin C/D treatment was not associated with better OS except in patients with AML with NPM1 mutations.

Full article: https://doi.org/10.1182/bloodadvances.2023010559

Transplantation

A survey of fully haploidentical hematopoietic stem cell transplantation in adults with high-risk acute leukemia: a risk factor analysis of outcomes for patients in remission at transplantation: Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is an alternative treatment to patients with high-risk acute leukemia lacking a human leukocyte antigen-matched donor. An analysis comprising 173 adults with AML and 93 with ALL who received a haplo-HSCT in Europe. All grafts were T cell–depleted peripheral blood progenitor cells from a direct family or other related donor. The results show that haplo-HSCT can be an alternative option for the treatment of high-risk acute leukemia patients in remission, lacking a human leukocyte antigen-matched donor. (ref: https://doi.org/10.1182/blood-2008-02-140095)

BPDCN

Multicenter analysis of outcomes in blastic plasmacytoid dendritic cell neoplasm offers a pretargeted therapy benchmark

  • Outcomes in a multicenter BPDCN population in the modern era provide a benchmark before targeted therapy.

  • Age <60 years, normal karyotype, and TdT positivity were associated with improved survival; pralatrexate and enasidenib had activity in BPDCN.

Full article: https://doi.org/10.1182/blood.2019001144

Long-term survival following autologous and allogeneic stem cell transplantation for blastic plasmacytoid dendritic cell neoplasm

We sought to clarify the role of high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (auto-HSCT) and allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN). We retrospectively identified 25 BPDCN patients (allo-HSCT, n = 14; auto-HSCT, n = 11) from registry data of the Japan Society for Hematopoietic Cell Transplantation and analyzed clinicopathologic data and clinical outcomes after transplantation. The median age at HSCT was 58 years (range, 17-67 years). All 11 patients who underwent auto-HSCT were in the first complete remission (CR1). With a median follow-up of 53.5 months, the overall survival rates at 4 years for patients who underwent auto-HSCT and allo-HSCT were 82% and 53% (P = .11), respectively, and progression-free survival rates were 73% and 48% (P = .14), respectively. Auto-HSCT for BPDCN in CR1 appears to provide promising results and deserves further evaluation in the setting of prospective trials. (Ref: https://doi.org/10.1182/blood-2015-01-621268)

Stem cell transplantation can provide durable disease control in blastic plasmacytoid dendritic cell neoplasm: a retrospective study from the European Group for Blood and Marrow Transplantation

Patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) have a poor prognosis with conventional chemotherapy. In the present study, we retrospectively analyzed the outcome of patients with BPDCN who underwent allogeneic stem cell transplantation (allo-SCT) or autologous stem cell transplantation (auto-SCT). A total of 39 patients (allo-SCT, n = 34; auto-SCT, n = 5) were identified in the European Group for Blood and Marrow Transplantation registry. The 34 allo-SCT patients had a median age of 41 years (range, 10-70) and received transplantations from sibling (n = 11) or unrelated donors (n = 23) between 2003 and 2009. MAC was used in 74% of patients. Nineteen allo-SCT patients (56%) received transplantations in first complete remission. The 3-year cumulative incidence of relapse, disease-free survival, and overall survival was 32%, 33%, and 41%, respectively. By univariate comparison, being in first remission at allo-SCT favorably influenced survival, whereas age, donor source, and chronic GVHD had no significant impact. We conclude that high-dose therapy followed by allo-SCT from related or unrelated donors can provide durable remission even in elderly patients with BPDCN. However, it remains to be shown if graft-versus-malignancy effects can contribute significantly to BPDCN control after allo-SCT. (Ref:https://doi.org/10.1182/blood-2012-08-448613)

More articles in BPDCN: https://ashpublications.org/search-results?q=BPDCN&fl_SiteID=1&page=1&qb={%22q%22:%22BPDCN%22}&utm_source=sfmc&utm_medium=email&utm_campaign=Innovations+in+BPDCN-+12-18-23&utm_term=https%3a%2f%2fashpublications.org%2fsearch-results%3fq%3dBPDCN%26fl_SiteID%3d1%26page%3d1%26qb%3d%7b%2522q%2522%3a%2522BPDCN%2522%7d&utm_id=298868&sfmc_id=19426595

Telehealth

Telehealth serious illness care program for older adults with hematologic malignancies: a single-arm pilot study

Older patients with AML and MDS feel shocked and bewildered when diagnosed. Serious illness conversations (SICs) may increase disease understanding and preparations for the future. However, SICs often happen late, in part because of clinician-perceived patient discomfort. Telehealth may promote patient comfort by allowing SICs to take place at home. This study assesses the feasibility and usability of a telehealth-delivered Serious Illness Care Program (SICP) for older adults with AML and MDS. We conducted a single-arm pilot study including 20 older adults with AML and MDS. Feasibility was measured using retention rate, with >80% considered feasible. Usability was measured using telehealth usability questionnaire (TUQ; range, 1-7): >5 considered usable. We collected other outcomes including acceptability and disease understanding and conducted post-visit qualitative interviews to elicit feedback. Hypothesis testing was performed at α = 0.10 owing to the pilot nature and small sample size. Retention rate was 95% (19/20); mean TUQ scores were 5.9 (standard deviation [SD], 0.9) and 5.9 (SD, 1.1) for patients and caregivers, respectively. We found the SICP to be acceptable. The majority of patients found the SICP to be very or extremely worthwhile (88.2%; 15/17), and reported it increased closeness with their clinician (75.0%; 12/16). After their visit, patient estimates of curability, and overall life expectancy aligned more closely with those of their clinicians. In qualitative interviews, most patients said that they would recommend this program to others (89.5%, 17/19). This study demonstrated that delivery of the telehealth SICP to older patients with AML and MDS is feasible, usable, and acceptable. (ref: https://doi.org/10.1182/bloodadvances.2023011046)

More

Recent articles in leukemia: leukemia | Page 1 | Search Results | American Society of Hematology (ashpublications.org)

Highlights from ASH

From 08th to 12th December 2023, ALAN attended the 65th ASH Annual Meeting and Exposition in San Diego (USA).

Reports on sessions

Latest treatment options for patients with relapsed, refractory or persistent AML

A comprehensive discussion on the available treatment options for acute myeloid leukemia (AML) and how to choose the most appropriate, unfolded during this educational session at ASH 2023. Dr Jacqueline Cloos from VU University Medical Center, Amsterdam, the Netherlands, discussed the challenges of categorising patients into different risk groups. The European Leukemia Network (ELN) risk classification that takes into account molecular and chromosomal abnormalities, was highlighted as an effective way to divide patients into favourable, intermediate, and adverse categories. Dr Cloos further underscored the clinical significance of measurable residual disease (MRD) and its pivotal role in treatment decisions, presenting evidence linking MRD negativity to improved outcomes. The presentation explored how and when to use various MRD assessment tools (quantitative real-time polymerase chain reaction [qRT-PCR], flow cytometry, next-generation sequencing, digital PCR) according to each patient’s risk classification and clinical picture. Important ongoing trials were discussed that aim to establish MRD as a substitute measure for treatment success and others that explore MRD-guided treatments. Dr Cloos concluded by highlighting the need for real-world evidence to complement the results from clinical trials and to demonstrate the value of MRD not just in the research setting.

The session continued with Dr Marion Subklewe from Ludwig-Maximilians-University Hospital, Munich, Germany, discussing the recent immunotherapy advancements for AML and their potential alongside established therapies to enhance MRD negativity and outcomes. The presentation covered various immunotherapies, such as antibody-drug conjugates, monoclonal antibodies, bispecifics and CAR T. Their challenges including identifying suitable target antigens and dealing with antigen escape- when tumor cells evolve to express fewer target antigens, reducing the ability of CAR T-cells to recognise and attack them effectively- were also discussed. Dr Subklewe concluded with a plea for reaching a consensus in assessing patient genetics, phenotype, and inflammatory signatures to tailor immunotherapy for individual AML patients and thus improve outcomes.

The session concluded with Dr Alison Walker from the Moffitt Cancer Center, Florida, USA, shifting into new treatments for patients who are relapse/refractory to initial therapies. Dr Walker explored factors that could be predictive of treatment failure, like the genetic mutation TP53, which has been linked to treatment resistance and those associated with positive responses (NPM1, IDH1, IDH2, DNMT3A mutations). Moreover, the importance of MRD assessment and how it can be a useful indicator of treatment response duration was noted. Dr Walker further explored the biological mechanisms behind resistance to venetoclax-based treatments seen in some patients. The speaker highlighted the scarcity of data for these patients who relapse during or after venetoclax plus hypomethylating agent therapy, emphasising the huge challenge in treatment and outcome improvement. Potential therapies for this patient group were then explored, including ongoing clinical trials, with novel combination therapies blocking more than one tumor target (e.g., BCL2, MCL1, menin) and immunotherapies like pivekimab sunirine (IMGN632) and magrolimab being highlighted as promising avenues. Nevertheless, Dr Walker stressed that relapse/refractory AML, regardless of initial therapy, remains an unmet therapeutic need and ongoing research to advance treatment options remains paramount.

What does this mean for patients?

Relapse or refractory AML remains an unmet therapeutic need. The session acknowledges the complexities involved in managing and treating this population. However, what stands out is the commitment to finding effective solutions for these patients with many novel combination therapies and immunotherapies being evaluated in clinical trials. Along with those, the current array of tools to assess MRD and to guide treatment decisions from the beginning based on each patient’s molecular and genetic abnormalities instils optimism for a more personalised approach that enhances patient outcomes.

The evolution of AML therapy: from tradition to innovation

In this educational session during ASH 2023, the evolution of therapies for acute myeloid leukemia (AML) was discussed along with how it has transformed the current standard of care. Dr Christoph Röllig, Universitätsklinikum TU Dresden, Germany, opened the discussion with a historical perspective on the use of intensive chemotherapy in AML. He highlighted that today’s real-world data demonstrate that intensive chemotherapy leads to significantly increased survival and longer freedom from disease. The session further delved into the comparison of intensive chemotherapy and newer agents like azacitidine (AZA) plus venetoclax (VEN). The superiority between these approaches is yet to be determined, pending the results of ongoing clinical trials. Dr Röllig presented evidence from trials on novel drugs like gemtuzumab ozogamicin, midostaurin, and quizartinib, which when combined with intensive chemotherapy show positive impacts on survival outcomes in specific AML patient groups. Exploring the future of intensive chemotherapy, Dr Röllig envisioned a future where novel agents complement or replace components of intensive chemotherapy, allowing for personalised and more effective treatments, guided by patient risk classification.

Dr Tara Lin from The University of Kansas Medical Center, Kansas, USA, focused on the combination of AZA and VEN for older and unfit patients, specifically discussing the VIALE-A trial. Updated data from this pioneering clinical trial demonstrate a consistent survival advantage over time for patients who received the AZA-VEN combination. This combination proves valuable, extending treatment options to elderly patients previously deemed unfit for therapy. The speaker discussed the advantages of a community and academic partnership in treating AML patients, emphasising the collaborative efforts required for comprehensive care, diagnostic testing, and access to clinical trials. The presentation delved into several retrospective studies examining the use of AZA-VEN in different patient populations, such as those with favorable risk cytogenetics or specific genetic mutations. The studies suggested potential benefits for certain subgroups but emphasised the need for prospective, randomised trials. Limited data on using AZA-VEN as a pre-transplant regimen was also discussed.

Concluding the session, Dr Justin Watts from the University of Miami Miller School of Medicine, Florida, USA, discussed the future of AML therapy with a focus on understanding mechanisms of VEN resistance and exploring targeted therapies for patients with specific genetic mutations (e.g., FLT3, TP53, IDH1/2). Dr Watts unraveled the intricacies of VEN resistance, outlining specific genetic mutations (e.g., FLT3, RAS, TP53, BAX) and cellular alterations contributing to it. Survival in AML is closely linked to specific mutations, with patients lacking FLT3, RAS, and TP53 mutations having better outcomes. With regards to targeted therapies, the speaker presented promising developments with IDH1 (ivosidenib) and IDH2 inhibitors (enasidenib) suggesting their potential addition to primary treatment along with VEN and AZA. For patients with FLT3 mutations, combining VEN with FLT3 blockers showed promise, especially in younger patients aiming for a transplant. Menin blockers, which could be a game-changer by targeting certain genetic mutations (MPM1, MLL rearrangements) were also discussed. Challenges remain, particularly in addressing treatment decisions in patients with RAS mutations or TP53 alterations due to mixed successes in clinical trials.

What does this mean for patients?

Overall, these advancements bring optimism and expand treatment options for individuals with AML. Increased survival benefits with intensive chemotherapy have now been confirmed in the real-world setting and newer combinations like AZA plus VEN, have shown promising outcomes particularly for older and unfit patients. Ongoing trials aim to determine the best approach, offering hope for more personalised and effective treatments. The discussion and current research on targeted therapies for specific genetic mutations signals a future with more tailored therapies and improved outcomes for AML patients.

Navigating challenges in AYA ALL: Insights from ASH 2023

The management of acute lymphoblastic leukemia (ALL) in adolescents and young adults (AYA) poses a unique challenge, given their age range of 15–39 years, placing them at the crossroads of pediatric and adult oncology. Despite improved outcomes in pediatric cases, AYA patients show less progress leading to what is commonly known as the “AYA age gap”. This ASH 2023 educational session explored treatment approaches and distinctions between AYA and pediatric patients with ALL and explored ways of enhancing outcomes within this patient group.

Dr John Molina from Taussig Cancer Institute, Ohio, USA, initiated the discussion by critically examining outcomes for AYA patients with B-cell ALL undergoing treatment with conventional pediatric or “pediatric-inspired” regimens as initial therapy. Critical evaluation of clinical trials showed that treatment location (adult or pediatric facilities) significantly impacted patient survival rates, with pediatric-inspired regimens leading to superior outcomes and emerging as a global standard for AYA ALL (CalGB10403 or Dana-Farber regimens). The integration of immunotherapies into established chemotherapy protocols, and the changing landscape of using hematopoietic stem cell transplant (HSCT) as a consolidative therapy for AYA patients who achieve a first complete remission were also discussed. Dr Molina concluded by stressing the importance of considering long-term toxicities, fertility concerns, and quality of life in AYA patients, along with employing next-generation sequencing (NGS) for the assessment of measurable residual disease (MRD) to guide treatment.

Dr Tamara Miller from the Children’s Healthcare of Atlanta, Georgia, USA, discussed the biological differences between pediatric and AYA patients, including variations in cancer biology, response to chemotherapy and the prevalence of specific genetic abnormalities. AYA patients may experience higher toxicities and lower tolerance to chemotherapy compared to younger patients. This is attributed to physiological changes during the AYA period, resulting in increased rates of adverse events and potential challenges in adhering to treatment. The need for increased care during this critical age for developing independence and identity, along with factors like fertility concerns, financial burdens, and disruptions in relationships, were highlighted as key challenges in AYA patients that could potentially influence adherence to therapy and increase the risk of relapse. Additionally, AYA patients seem less likely to enroll in clinical trials, with various barriers including the lack of open trials for this age group, mismatched age requirements, and competing priorities such as school and work, contributing to this.

Dr Emily Curran, University of Cincinnati, Ohio, USA, concluded the session by highlighting novel therapeutic advancements and challenges in AYA patients. Recognising challenges posed by adverse cancer biology prevalent in some AYA cases (e.g., Philadelphia chromosome[pH]-like ALL, KMT2A rearranged ALL), Dr Duran discussed ongoing investigations, including kinase inhibitors for pH-like ALL and menin inhibition for KMT2A ALL. Dr Curan mentioned the lack of immunotherapy options for T-cell ALL, unlike B-cell ALL, emphasised ongoing trials exploring BCL2 and BCLXL blockade and highlighted early promise of CAR T-cell therapy in this patient group. The talk addressed the psychosocial factors affecting AYA patients, including treatment adherence issues, health insurance, and clinical trial enrollment. The use of technology, such as text message reminders, as a potential strategy to improve adherence and clinical trial enrollment were also noted.

What does this mean for patients?

The session underscored the unmet needs of AYA patients with ALL, stressing the necessity for interventions addressing the unique cancer biology, genetics, and psychosocial aspects. Comprehensive strategies targeting biological, treatment-related, psychosocial, and enrollment challenges are crucial to enhance outcomes and advance clinical knowledge in this patient group. Bridging the “AYA age gap” remains an ongoing journey.

How to choose between stem cell transplantation and CAR T-cell therapy in pediatric ALL

An educational session took place at ASH 2023 focusing on the treatment of pediatric acute lymphoblastic leukemia (ALL). Most children with ALL achieve long-term remission through chemotherapy alone, but some relapse or remain refractory, leading to the need for additional therapies. For these high-risk patients, two key treatments have shown great promise: hematopoietic stem cell transplantation (HSCT) and CAR T-cell therapy. HSCT has a long-established track record of favorable survival rates, whilst CAR T has shown transformative potential with stellar remission rates. To date, the decision between HSCT and CAR T is primarily driven by physician judgment and institutional preference. The session explored the criteria used to guide this choice with Dr Nirali Shah from the National Institutes of Health, outlining individual patient risk factors, prior complications, organ functions, donor availability, institutional expertise, and access to cellular therapy as key considerations. Moreover, Dr Shah discussed delayed toxicities following CAR T, including effects on neurocognition and fertility and their management. She emphasised the need for research evaluating other late CAR T toxicities, including the monitoring for potential second tumors and future fertility.

Previously vital for chemotherapy-resistant cases, HSCT faces competition from CAR T-cell therapy. Dr Alice Bertaina from Stanford University, California, USA, further discussed the need to redefine the timing and use of HSCT in pediatric ALL. Dr Bertaina concluded that HSCT remains a crucial treatment for high-risk and relapse-refractory ALL patients and that choosing the right treatment involves considering various factors unique to each patient and the treatment center, beyond the reported literature. Proposing a future strategy, Dr Bertaina advocated for the integration of HSCT with CAR T in this high-risk population, tailored by specific patient risk factors. She further stressed the need to design clinical trials focused on finding biomarkers that could help guide this decision in this rare high-risk pediatric population to improve patient outcomes.

Dr Stephen Gosselk from St. Jude Children’s Research Hospital, Tennessee, USA, concluded the session by addressing the challenges of determining the optimal step after CAR T-cell therapy. He acknowledged the absence of a clear treatment algorithm for deciding which patients should undergo HSCT, whilst exploring ways to prevent relapse after CAR T in pediatric ALL. He also stated that the decision to proceed to HSCT involves the evaluation of multiple factors like, prior therapies, residual disease, genetic risk, conditioning regimens, as well as CAR T product characteristics. Dr Gosselk highlighted key findings indicating that achieving no residual disease, sustaining low levels of B-cells for six months, administering an optimal dose of the conditioning regimen, and ensuring a high number of circulating CAR T-cells post-infusion are pivotal indicators of favorable outcomes. In conclusion, the ongoing requirement for predictive biomarkers and prevention of CAR T relapse were highlighted. Acknowledging the need for additional research, the speaker emphasised the importance of improving CAR T products to minimise challenges associated with low persistence within the body or antigen escape- when tumor cells evolve to express fewer target antigens, reducing the ability of CAR T-cells to recognise and attack them effectively.

What does this mean for patients?

This educational session on pediatric ALL, instilled optimism regarding treatment options for high-risk patients facing relapse or unresponsiveness to chemotherapy. Beyond traditional HSCT, CAR T offers a valuable alternative, prompting the need to redefine criteria for choosing between the two. The speakers highlighted the ongoing establishment of these criteria, with residual disease, individual risks, prior therapies/toxicities, treatment availability, and center expertise being key considerations for decision-making. The importance of an integrated approach with both treatments used and of novel clinical trials investigating biomarkers to help guide treatment decisions were highlighted.

Fertility empowerment for hematologic cancer patients: Navigating challenges and options

Patients with hematologic cancers encounter a myriad of challenges when considering fertility preservation options. With the advent of innovative technologies and a heightened awareness of the importance of fertility preservation, numerous opportunities have emerged. Dr Erica Marsh from the University of Michigan, Michigan, USA, opened the session by reflecting on the current design of health systems, urging an understanding of who these systems include or exclude. The speaker provided definitions for key terms like inequality, equality, and equity, in the context of healthcare. The need for tools to address health disparities revolving around race, ethnicity, socioeconomic status, gender, age, mental health and historical discrimination was also highlighted. Connecting health disparities to infertility, Dr Marsh encouraged the understanding of patients through two key concepts: i) intersectionality, recognising individuals as multifaceted beings influenced by race, gender, reproductive status, occupation, and more; and ii) positionality, how our worldview is shaped by social and cultural influences, determining how we perceive and engage with the world.

Dr Alison Loren from the University of Pennsylvania, Pennsylvania, USA, continued the discussion by highlighting the emotional impact of infertility on cancer survivors and urging for early patient-doctor discussions even if immediate fertility preservation is not deemed feasible. Dr Loren addressed existing barriers that include provider discomfort, racial and socioeconomic biases, and insurance coverage issues. Fertility options for critically ill patients who cannot undergo standard procedures were also explored, with a focus on gonadotropin-releasing hormone analogs, which remain controversial and ovarian tissue cryopreservation. The speaker further emphasised the importance of ongoing fertility discussions post-therapy, revealing the risk of premature ovarian failure in survivors. Dr Loren concluded with a plea to hematologists to initiate fertility conversations, be aware of biases, establish connections with reproductive specialists, and consider legislative advocacy for better patient coverage.

Dr Mindy Christianson from the Cleveland Clinic, Cleveland, Ohio, USA, concluded the session by addressing surgical fertility preservation, emphasising ovarian tissue transplantation and introducing a novel technique called uterine suspension. Ovarian tissue cryopreservation, pioneered in 2000, has evolved, with over 200 reported live births. Standard practice involves removing one ovary for freezing and potential future transplantation. Various techniques exist for transplanting tissue, but there is no standardised best practice. The talk highlighted the challenges and success rates associated with different methods. Switching focus to uterine preservation after pelvic radiation, the speaker discussed uterine fixation, a surgical technique which involves moving the uterus out of the radiation field for protection. While still new, this approach shows promise in preserving fertility for cancer patients. The discussion concluded with tips for success in fertility preservation centers, emphasising the need for open communication between oncologists and fertility specialists. Building a patient-centered team that is well-versed in all pathways to parenthood, including in-vitro fertilization (IVF), egg donation, and gestational surrogacy, was stressed. Additionally, the importance of healthcare professionals being aware of financial considerations, hurdles, and state mandates for fertility coverage was highlighted.

What does this mean for patients?

This information empowers hematologic cancer patients to consider and address fertility concerns, urging early discussions with healthcare providers and reproductive specialists. The emotional impacts and barriers survivors face are slowly being acknowledged and together with the evolving techniques, from ovarian tissue transplantation to uterine preservation, they offer hope for improved fertility preservation. A patient-centric approach with open communication and good awareness of parenthood pathways from healthcare professionals are crucial for informed decision-making.

Advancing patient-centric care for hematological malignancies

Dr Dipty Patel-Donnelly from Virginia Cancer Specialists, Virginia, USA, led an educational session during ASH 2023, addressing the management of high-risk hematological malignancies in the community. The session covered acute leukemias, complicated lymphomas, multiple myeloma, and clinical research in the community. The speaker highlighted the unique challenges of acute leukemias and complicated lymphomas with case study examples, emphasising their rarity and acute presentation. The discussion covered the complexities of treatments and the coordination needed for transfusions. Despite challenges, advancements in novel therapies offer hope, but their application in the community setting requires overcoming logistical barriers. The significance of maintaining close communication, frequent patient visits, and a comprehensive care team, including social work and nutritionists, was a key highlight for managing these patients. Through a case example, the speaker also highlighted the limitations of available therapies and clinical trials in the community and how providing local access to innovative treatments can significantly improve patient outcomes. Dr Patel-Donnelly proposed a collaborative model between academic and community settings through accessing regional experts, early education programs, and co-management strategies to help improve patient outcomes, reduce costs, and increase engagement in clinical trials.

Dr Jesus Berdeja, Sarah Cannon Research Institute, Tennessee, USA, emphasised the importance of improving patient access, inclusion, and the overall complexity of care by decentralising advanced therapies and bringing them closer to patients in local clinics. Drawing insights from myeloma treatment experiences, the speaker highlighted the continuous and multi-faceted nature of the patient journey, which often requires a dynamic interplay between local and specialised clinics.

Access to advanced therapies, with a focus on CAR T and bispecific antibodies, was also a focus of the discussion. While CAR T shows great promise for hematological malignancies, it poses challenges related to patient selection, the necessity for bridging, and limited availability. On the other hand, bispecifics are considered more forgiving, with fewer logistical challenges, making them potentially suitable for older or frail patients. The speaker urged community practices to assess their capabilities for administering transplantation, CAR T and bispecifics and suggested internal referrals within practices and prompt consideration of administering bispecifics locally or through external referrals with the aim of bringing these advanced therapies closer to community patients.

Lastly, Dr Ruemu Birhiray, Hematology Oncology of Indiana, Indiana, USA, discussed the importance of diversity in clinical trials, particularly in the context of community-based practices. He highlighted disparities in care due to limited access to clinical trials, emphasising the challenges faced by patients who are unwilling or unable to travel to academic centers. Dr Birhiray presented a case of a patient who, despite having access to a clinical trial, opted for treatment locally due to caregiving responsibilities. The speaker addressed structural barriers, including the historical lack of diversity in clinical trials, and advocated for intentional efforts to improve inclusivity. Although acknowledging the National Cancer Institute initiatives to bring research to community practices, the current disparities in the availability of innovative therapies at community sites was pointed out. The need for individual responsibility among healthcare professionals to enroll diverse populations in clinical studies was stressed along with the need for a comprehensive and collaborative practice approach.

What does this mean for patients?

The discussions about community management at ASH 2023 promise positive shifts for patients with hematological malignancies, aiming for increased accessibility to advanced therapies like CAR T and bispecifics in local clinics. Collaborative care approaches recognise the ongoing nature of patient journeys, aiming for individualised treatments, inclusive clinical trials, and academic-community collaborations to provide more accessible and inclusive care, ultimately improving patient outcomes for all.

From HARMONY project

Machine Learning Provides Individualized Prediction of Outcomes after First Complete Remission in Adult AML Patients – Results from the HARMONY Big Data Platform 

A model providing individualized outcome estimations in adult AML patients with intensive treatment approaches was developed and validated. Predictions for relapse-free survival, cumulative incidence of relapse and overall survival were more accurate than those in the ELN2022 risk stratification. The model is accessible online via an interactive web calculator, and with further validation and refinement, it could be used in the future for clinical decision-making.

Outcome of Intensively Treated Elderly AML Patients Reported to the Harmony Alliance Compares Well to Outcome of Control Patients of the Prospective Randomized HOVON 103 Study in Elderly AML

The evaluation of novel drugs in hemato-oncology is hampered by the relatively large sample sizes needed in RCTs. Supplementing trial data with external control data could overcome this obstacle. Our comparative study revealed that HARMONY Alliance data matched RCT data well for overall survival. Matched HARMONY Alliance data could therefore supplement prospectively collected control data in studies evaluating intensive therapy in elderly AML.

Long-Term Outcome of 1296 Patients with Newly Diagnosed with APL: A HARMONY Alliance Study

Acute promyelocytic leukemia (APL) is now curable in 75-90% of patients using targeted agents. RCTs revealed similar patient outcomes for chemotherapy and non-chemotherapy treatment regimens. However, the reliability of these outcomes was limited by the small patient cohorts used. Our long-term analysis using the larger HARMONY APL registry patient cohort revealed a significant survival advantage for the non-chemotherapy regimen with reduced early death rates and prolonged overall survival independent of Sanz risk score.

Other data published

Obecabtagene Autoleucel (obe-cel, AUTO1) for Relapsed/Refractory Adult B-cell Acute Lymphoblastic Leukemia (R/R B-ALL): Pooled Analysis of the Ongoing FELIX Phase Ib/II Study – Autolus

Long-term Efficacy and Safety of Obecabtagene Autoleucel (obe-cel) in Adult Patients (pts) with Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia ([R/R B-ALL]; pooled analysis of ALLCAR19 and FELIX Phase Ib Studies) or Other B-cell Malignancies (ALLCAR19 Extension Study) – Autolus

Delivery of Obecabtagene autoleucel (obe-cel, AUTO1) for the FELIX Pivotal Study Demonstrating Robust Cell Processing, Robust Release Testing, and Reliable Logistics, Together with Readiness for Sustainable Patient (pt) Care – Autolus

Patient-Reported Outcomes in Acute Myeloid Leukemia Patients with FLT3-ITD Mutation Receiving Quizartinib Vs. Standard Chemotherapy: Results from the Quantum-First Trial – Daiichi-Sankyo

Quantum-First: Safety By Treatment Phase and By Age in Newly Diagnosed (nd) Patients (pts) with FMS-like Tyrosine Kinase 3–Internal Tandem Duplication (FLT3-ITD) Positive Acute Myeloid Leukemia (AML) – Daiichi – Sankyo

Ponatinib Versus Imatinib in Patients with Newly Diagnosed Ph+ ALL: Subgroup Analysis of the Phase 3 PhALLCON Study – Incyte

Use of ponatinib alone or combined with other therapies in relapsed/refractory Ph-like acute lymphoblastic leukemia. A  Campus ALL real-life study – Incyte

Comparison between dasatinib-blinatumomab vs ponatinib-blinatumomab chemo-free strategy for newly diagnosed Ph+ acute lymphoblastic leukemia patients. Preliminary results of the GIMEMA ALL2820 trial – Incyte

A First-in-Human Phase 1 Study of the Menin-KMT2A (MLL1) Inhibitor JNJ-75276617 in Adult Patients with Relapsed/Refractory Acute Leukemia Harboring KMT2A or NPM1 Alterations – Janssen

Optimizing Outcomes with Myeloablative Conditioning in Older Patients: Efficacy and Safety of Precision Engineered Orca-T in Patients > 55 Years Old with Hematologic Malignancies – Orcabio

Safety and Efficacy of Orca-Q with Haploidentical Donors for the Treatment of Advanced Hematologic Malignancies without the Use of Post-Transplant Cyclophosphamide – Orcabio

Phase 1 Trial Results for Patients with Advanced Hematologic Malignancies Undergoing Reduced Intensity Allogeneic HCT with Orca-T Donor Cell Therapy Product and Single Agent Tacrolimus – Orcabio

Molecular Measurable Residual Disease in Patients with Newly Diagnosed mIDH1 Acute Myeloid Leukemia Treated with Ivosidenib + Azacitidine – Servier

Real-World Treatment Patterns and Clinical Outcomes in Newly Diagnosed Acute Myeloid Leukemia with and without mIDH1 Treated with Intensive Chemotherapy from an International Real-World Database (REAL-IDH) – Servier

A Comparison of Acute Myeloid Leukemia Regimens: Hypomethylating Agents Combined With Ivosidenib or Venetoclax in Newly-diagnosed Patients With IDH1 Mutations: A Real-world Evidence Study – Servier

Trial in Progress: An Open Label Phase I/II, Multicentric Study Evaluating Safety, Pharmacokinetics and Efficacy of S65487, a BCL-2 inhibitor Combined with AZA in Adults with Previously Untreated AML Ineligible for Intensive Treatment – Servier

Ivosidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH1 – Servier

Assessment of Outcomes of Consolidation Therapy by  Number of Cycles of Blinatumomab Received in Newly Diagnosed Measurable Residual Disease Negative Patients with B-lineage Acute Lymphoblastic Leukemia: in the ECOG-ACRIN E1910 Randomized Phase III National Clinical Trials Network Trial – Servier

The Impact of Age and Genomics on Drug Sensitivity in 1,076 Children and Adults with B-cell Acute Lymphoblastic Leukemias – Servier

Minimal Residual Disease-Negative Complete Remission at the End of Induction is a Prognostic Indicator of Long-Term Survival in Adult Patients with Ph+ Acute Lymphoblastic Leukemia Receiving First-Line Therapy – Takeda

Chemotherapy-Free Combination of Blinatumomab and Ponatinib in Adults with Newly Diagnosed Philadelphia Chromosome–Positive Acute Lymphoblastic Leukemia: Updates from a Phase II Trial – Takeda

A Phase II Study of Low-Intensity Chemotherapy (Mini-Hyper-CVD) and Ponatinib Followed by Blinatumomab and Ponatinib in Patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia – Takeda

Real-world outcomes of ponatinib treatment in 724 patients with CML and Ph+ ALL: a post-marketing surveillance study with a special interest in arterial occlusive events in Japan – Takeda (study sponsored by Otsuka)

ASH 2023

From 08th to 12th December 2023, Catherine, Jan and Samantha attended the 65th ASH Annual Meeting and Exposition in San Diego (USA).

Before leaving, we had the chance to meet virtually with our members and supporters who couldn’t attend the congress in person.

On Friday 08th December, Jan Geissler (interim ALAN Chair) and Samantha (ALAN Network Director) gave an update to ALAN Members and Supporters on the  2023 plans and on planned 2024 activities.  We are happy to share the presentation (if not already received) – just let us know by email at samantha@acuteleuk.org.

We met with our colleagues from other advocacy organizations at the Pfizer ASH Patient Advocacy breakfast and working session and were excited to present data from the carer global leukemia experience survey and share our perspective. 

On the sunday 10th December, we presented our poster “Understanding Relapsed/Refractory Acute Leukemia Patients Treatment Preferences“.

The congress was also the opportunity to meet with some of our members and sponsors but also our advocacy colleagues.

And this is what it feels like on a typical #ASH23 evening after 13.49km and 18.012 steps at the San Diego Conference Center, chasing between meetings and sessions all day.

Until we meet again, the ALAN team wishes you a wonderful holiday season !