Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group

Kröger, Nicolaus; Bacigalupo, Andrea; Barbui, Tiziano; Ditschkowski, Markus; Gagelmann, Nico; Griesshammer, Martin; Gupta, Vikas; Hamad, Nada; Harrison, Claire; Hernandez-Boluda, Juan Carlos; Koschmieder, Steffen; Jain, Tania; Mascarenhas, John; Mesa, Ruben; Popat, Uday R; Passamonti, Francesco; Polverelli, Nicola; Rambaldi, Alessandro; Robin, Marie; Salit, Rachel B; Schroeder, Thomas; Scott, Bart L; Tamari, Roni; Tefferi, Ayalew; Vannucchi, Alessandro M; McLornan, Donal P; Barosi, Giovanni

doi:10.1016/s2352-3026(23)00305-8

Cited by 17 publications

(15 citation statements)

References 73 publications

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“…Engraftment, rates of graft-vs. -host disease (GvHD), progression-free survival (PFS), and OS comparable to those reported in younger patients were shown in selected patients with primary or secondary myelofibrosis aged 60 to 78 years old who received HSCT from Human Leukocyte Antigen (HLA)-identical siblings or unrelated donors ( 21 ). Thus, age should not represent an absolute contraindication to allo-HSCT, in case of absent or well-controlled comorbidities ( 6 ).…”

Section: Age At Transplantmentioning

confidence: 99%

“…The use of RIC regimens allow more favorable survival in older patients aged > 65 years old with no or minimal comorbidities ( 22 ). EBMT/ELN recommendations suggest considering the possibility of HSCT case by case, taking into account patients’ and disease variables and also the recipient’s preferences ( 6 ).…”

Section: Age At Transplantmentioning

confidence: 99%

“…To ensure the most complete evaluation of disease-associated risk, a simultaneous assessment of as many scores as possible could be facilitated by a PMF-specific calculator ( 45 ). However, recent EBMT/ELN recommendations indicate allogeneic HSCT based exclusively on DIPSS, MIPSS70, and MIPSS70 plus scores ( 6 ).…”

Section: Selection Of Patientsmentioning

confidence: 99%

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Myelofibrosis and allogeneic transplantation: critical points and challenges

Ranalli,

Natale,

Guardalupi

et al. 2024

Front. Oncol.

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New available drugs allow better control of systemic symptoms associated with myelofibrosis (MF) and splenomegaly but they do not modify the natural history of progressive and poor prognosis disease. Thus, hematopoietic stem cell transplantation (HSCT) is still considered the only available curative treatment for patients with MF. Despite the increasing number of procedures worldwide in recent years, HSCT for MF patients remains challenging. An increasingly complex network of the patient, disease, and transplant-related factors should be considered to understand the need for and the benefits of the procedure. Unfortunately, prospective trials are often lacking in this setting, making an evidence-based decision process particularly arduous. In the present review, we will analyze the main controversial points of allogeneic transplantation in MF, that is, the development of more sophisticated models for the identification of eligible patients; the need for tools offering a more precise definition of expected outcomes combining comorbidity assessment and factors related to the procedure; the decision-making process about the best transplantation time; the evaluation of the most appropriate platform for curative treatment; the impact of splenomegaly; and splenectomy on outcomes.

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Section: Age At Transplantmentioning

confidence: 99%

Section: Age At Transplantmentioning

confidence: 99%

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Myelofibrosis and allogeneic transplantation: critical points and challenges

Ranalli,

Natale,

Guardalupi

et al. 2024

Front. Oncol.

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mentioning

confidence: 99%

“…2 It is therefore critical to accurately assess transplantation risk and estimate survival with medical therapies to determine the most appropriate treatment approach for each individual. 3 Several prognostic models are available to categorize patients into risk groups. [4][5][6][7][8][9][10] Despite their utility, these models have limitations, such as exclusive applicability to specific MF subtypes, the need for karyotypic analysis, which may be challenging due to insufficient bone marrow aspiration, or reliance on Next Generation Sequencing (NGS) techniques that may not be widely accessible.…”

mentioning

confidence: 99%

Integrating AIPSS‐MF and molecular predictors: A comparative analysis of prognostic models for myelofibrosis

Mosquera‐Orgueira,

Arellano‐Rodrigo,

Garrote

et al. 2024

HemaSphere

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New drugs in myelofibrosis: Critical assessment of additional value to monotherapy with JAK inhibitors

Gangat,

Tefferi

2024

American J Hematol

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Among the JAK2 mutation-prevalent myeloproliferative neoplasms, myelofibrosis (MF) is prognostically the worst with median survival estimates ranging from less than 2 years to over 10 years, depending on the presence or absence of specific genetic and clinical risk factors. 1 Moreover, quality of life (QoL) in MF is severely compromised by progressive anemia, splenomegaly, and constitutional symptoms. At present, the only treatment modality in MF with the potential to cure the disease or prolong survival is allogeneic hematopoietic stem cell transplant. 2 On the other hand, current drug therapy, including that with JAK inhibitors (JAKi), has been shown to be effective in improving QoL by way of alleviating constitutional symptoms, reducing spleen size, reversing cachexia, and, in some instances, resulting in resolution of red cell transfusion need. 3 Ruxolitinib was the first JAKi to receive the United States Food and Drug Administration (FDA) approval in 2011, followed by approval of three additional JAKi, namely, fedratinib (2019), pacritinib (2022), and momelotinib (2023), the latter two in the context of thrombocytopenia (platelet count <50 Â 10 9 /L), and anemia, respectively. 4 All four FDA-approved JAKi alleviate

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Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group

Cited by 17 publications

References 73 publications

Myelofibrosis and allogeneic transplantation: critical points and challenges

Myelofibrosis and allogeneic transplantation: critical points and challenges

Integrating AIPSS‐MF and molecular predictors: A comparative analysis of prognostic models for myelofibrosis

New drugs in myelofibrosis: Critical assessment of additional value to monotherapy with JAK inhibitors

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