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3678 Modified Delphi Process to Develop Best Practice Recommendations for Myelofibrosis Management in Routine Clinical Practice in the Asia-Pacific Region (The APAC-MF Alliance)

Program: Oral and Poster Abstracts
Session: 903. Health Services and Quality Improvement: Myeloid Malignancies: Poster II
Hematology Disease Topics & Pathways:
Clinical Practice (Health Services and Quality), Diseases, Treatment Considerations, Myeloid Malignancies
Sunday, December 8, 2024, 6:00 PM-8:00 PM

Harinder Gill, MBBS, MD, FRCP (Edin, Glasg, Lond), FRCPath, FHKCP, FHKAM (Medicine)1, Chul Won Choi2*, Hein Than, MD, FRCPath, MRCP3, Hsin-An Hou, MD, PhD, MSc4*, Chul Won Jung, MD5, Yok-Lam Kwong, MD6*, Garret M. K. Leung6*, Melissa G. M. Ooi7,8*, Ponlapat Rojnuckarin, MD9, David M. Ross10*, Lee-Yung Shih, MD11, Katsuto Takenaka12*, Winnie Z. Y. Teo, FRCPath, MRCP7,13* and Keita Kirito, MD14

1Department of Medicine, The University of Hong Kong, Hong Kong, Hong Kong
2Department of Internal Medicine, Korea University College of Medicine, Seoul, Korea, Republic of (South)
3Department of Haematology, Singapore General Hospital, Singapore, Singapore
4Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan
5Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea, Republic of (South)
6Department of Medicine, School of Clinical Medicine, LKS Faculty of Medicine, The University of Hong Kong, Hong Kong, Hong Kong
7Department of Hematology‑Oncology, National University Cancer Institute, Singapore, Singapore
8Department of Medicine, Yong Loo Lin School of Medicine, National University, Singapore, Singapore
9Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand
10Royal Adelaide Hospital and Flinders Medical Centre, Adelaide, SA, Australia
11Chang Gung Memorial Hospital‑Linkou, Chang Gung University, Taoyuan, Taiwan
12Department of Hematology, Clinical Immunology, and Infectious Diseases, Ehime University Graduate School of Medicine, Toon, Japan
13Fast and Chronic Program, Alexandra Hospital, Singapore, Singapore
14University of Yamanashi, Chuo, Japan

Background: While international treatment guidelines for myelofibrosis (MF) have contributed to the standardization of best clinical practice and patient care globally, a need remains in the Asia-Pacific (APAC) region for treatment recommendations that take into account local epidemiology, healthcare infrastructure, and resource availability. In response, a Steering Committee (SC) of expert hematologists from Asia and Australia developed consensus statements (CSs) to optimize MF treatment in the APAC region. Herein, we present updated results of the program following the final round of extended faculty (EF) voting.

Objectives: To develop APAC region-specific, evidence- and consensus-based recommendations for routine MF management, which offer practical strategies to support clinicians and guide therapeutic decision-making.

Methods: A modified Delphi methodology was used to draft and agree CSs in response to 13 clinical questions representing unmet needs in the management of patients with MF in the APAC region. An SC was formed of 14 expert APAC hematologists who identified these questions across four themes: 1) thresholds for anemia, and when to initiate or modify treatment; 2) when to initiate or modify treatment for thrombocytopenia; 3) Janus kinase inhibitor failure and what would warrant switching treatment; and, 4) appropriate risk stratification models for MF in the APAC region. Findings from a systematic literature review, along with clinical insights of the SC, were used to draft the CSs. An APAC EF, including expert hematologists and representatives from patient advocacy groups, voted on the CSs, with consensus reached when 75% of respondents agreed within a range of 7–9 on a 9-point scale (1=strongly disagree, 9=strongly agree). Voters scoring <7 were required to provide written rationale, used to refine the CSs for re-voting.

Results: After two voting rounds, all CSs reached a high level of consensus (12/13 in the range of 90–100% and 1/13 in the range of 80–90%). In the first voting round, 12/13 CSs reached consensus among voters (hematologists [n=57], and patient representatives from MPN-JAPAN [n=1] and MPN Alliance Australia [n=1]). The CS that did not reach consensus related to how MF management changes if thrombocytopenia evolves during treatment; EF feedback highlighted concerns about the efficacy, risk, and availability of treatments in the APAC region (including danazol, IMiDs, and eltrombopag). Eltrombopag was removed as a recommended treatment option due to limited access and supporting evidence, while danazol and IMiDs were retained. The SC also identified a further five CSs that would benefit from refinement, including CSs on anemia treatment and use of risk stratification models. For CSs under the anemia theme, feedback included concerns on access to treatment options (including erythropoiesis-stimulating agents, danazol, and thalidomide), the link between anemia and weight loss, and the lack of robust evidence and guidelines for iron chelation use. Revisions included removing ‘weight loss’ from symptoms to consider when evaluating the need for anemia treatment (fatigue, shortness of breath, and poor appetite were retained) and more specific wording around treatment options for anemia and iron overload. For the risk stratification theme, feedback emphasized the lack of reimbursement for next-generation sequencing-integrated models and the relative pros and cons of these models vs more widely used scores. Revisions included updates to a proposed algorithm for prognostic score selection in MF. A second voting round was held, and consensus was achieved for all six of the resubmitted CSs. This iterative process aimed to ensure broad consensus and recommendations that meet the needs of clinicians and patients in the APAC region.

Conclusions: The APAC-MF consensus program established a framework for addressing key challenges in MF care within the APAC region. Consensus was achieved after two voting rounds among both the APAC SC hematologists and EF members on strategies to address the multifaceted aspects of MF care. This program highlighted the importance of considering regional nuance and individualized therapy, and we believe that continued collaboration and refinement will enhance care quality across the APAC region. This work will be developed into a clinical resource to support healthcare professionals in the care of patients with MF in the APAC region.

Disclosures: Gill: Astellas: Consultancy, Honoraria, Other: Lecture fees, Research Funding; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Conference Support; Lecture fees, Research Funding; Imago Biosciences: Research Funding; Otsuka: Consultancy, Other: Conference Support; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees; Celgene: Research Funding; PharmaEssentia Corporation: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Conference Support, Research Funding; Pfizer: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Conference Support, Research Funding; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Conference Support; Lecture fees, Research Funding; MSD: Consultancy, Honoraria, Other: Conference Support; Lecture fees, Research Funding; Jacobson Pharma Corporation: Consultancy, Honoraria, Research Funding; GSK: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Lecture fees, Research Funding. Than: PharmaEssentia Corporation: Consultancy, Honoraria, Other: Advisory fees; BMS: Consultancy, Honoraria, Other: Advisory fees; GSK: Consultancy, Honoraria, Other: Advisory fees; AbbVie: Consultancy, Honoraria, Other: Advisory fees; Novartis: Consultancy, Honoraria, Other: Advisory fees. Hou: Panco healthcare Co: Consultancy, Honoraria, Other: Travel fees; Merck Sharp & Dohme: Consultancy, Honoraria, Other: Travel fees; Celgene: Consultancy, Honoraria, Other: Travel fees, Research Funding; Sandoz: Consultancy, Honoraria, Other: Travel fees; AbbVie: Consultancy, Honoraria, Other: travel fees, Research Funding; Astellas: Consultancy, Other: Travel fees, Research Funding; Ono: Consultancy, Honoraria, Other: Travel fees; Lotus: Consultancy, Honoraria, Other: Travel fees; PharmaEssentia: Research Funding; Johnson & Johnson: Consultancy, Honoraria, Other: Travel fees; Chugai: Consultancy, Honoraria, Other: Travel fees; IQVIA: Consultancy, Honoraria, Other: Travel fees; Eli Lilly: Consultancy, Honoraria, Other: Travel fees; CSL Behring: Consultancy, Honoraria, Other: Travel fees; Daiichi Sankyo: Consultancy, Honoraria, Other: Travel fees; PharmaEssentia: Consultancy, Honoraria, Other: Travel fees; Zuellig Pharma: Consultancy, Honoraria, Other: Travel fees; TTY Biopharm Company: Consultancy, Honoraria, Other: Travel fees ; Roche: Consultancy, Honoraria, Other: Travel fees; TSH Biopharm: Consultancy, Honoraria, Other: Travel fees; Takeda: Consultancy, Honoraria, Other: Travel fees; Pfizer: Consultancy, Honoraria, Other: Travel fees; Kirin: Consultancy, Honoraria, Other: Travel fees; BeiGene: Consultancy, Honoraria, Other: Travel fees; Novartis: Consultancy, Honoraria, Other: Travel fees; Synmosa: Consultancy, Honoraria, Other: Travel fees; AstraZeneca: Consultancy, Honoraria, Other: Travel fees; Kirin: Research Funding; BMS: Consultancy, Honoraria, Other: Travel fees, Research Funding; GSK: Honoraria. Jung: GSK: Honoraria. Kwong: Takeda: Consultancy; Novartis: Consultancy, Research Funding; Roche: Consultancy; Merck: Consultancy, Research Funding; Janssen: Consultancy; Celgene: Consultancy; BMS: Consultancy, Research Funding; BeiGene: Consultancy; Bayer: Consultancy; GSK: Honoraria; Astellas: Consultancy; Amgen: Consultancy. Leung: GSK: Honoraria. Ooi: BMS: Honoraria, Other: Advisory board; GSK: Honoraria; Janssen: Honoraria, Other: Advisory board; Antegene: Honoraria, Other: Advisory board; Amgen: Honoraria, Other: Advisory board; Steward Cross: Honoraria, Other: Advisory board. Ross: Novartis: Consultancy, Honoraria, Other: Advisory fees, Research Funding; Keros: Consultancy, Other: Advisory fees, Research Funding; Menarini: Consultancy, Other: Advisory fees; Takeda: Consultancy, Other: Advisory fees; Avance CRO: Consultancy, Other: Advisory fees; MSD: Research Funding; MorphoSys: Research Funding; Protagonist: Research Funding; Incyte: Research Funding; Sumitomo: Research Funding; GSK: Honoraria, Research Funding. Shih: GSK: Honoraria. Takenaka: PharmaEssentia Japan: Other: Lecture fees; GSK: Honoraria; Novartis: Other: Lecture fees. Teo: Novartis: Consultancy, Honoraria, Other: Advisory fees; GSK: Consultancy, Honoraria, Other: Advisory fees; PharmaEssentia Corporation: Consultancy, Honoraria, Other: Advisory fees; BMS: Consultancy, Honoraria, Other: Advisory fees. Kirito: PharmaEssentia Japan: Other: Lecture fees; GSK: Honoraria.

OffLabel Disclosure: Danazol: synthetic attenuated androgen. Off-label use: treatment of MF-related anemia and thrombocytopenia. Erythropoietin-stimulating agents (ESAs): recombinant versions of erythropoietin (EPO). Off-label use: treatment of MF-related anemia. Immunomodulatory drugs (IMiD®): cereblon modulators, including thalidomide and its analogues. Off-label use: treatment of MF-related anemia and thrombocytopenia. Ruxolitinib: JAK1/2 inhibitor. Off-label use: treatment of MF in patients with platelet counts <50x109/l. Corticosteroids: synthetic steroid hormones. Off-label use: treatment of MF-related thrombocytopenia.

*signifies non-member of ASH