Abstract
Moving from indication to transplantation is a critical process in myelofibrosis. Most of guidelines specifically focus on either myelofibrosis disease or transplant procedure, and, currently, no distinct indication for the management of MF candidates to transplant is available. Nevertheless, this period of time is crucial for the transplant outcome because engraftment, non-relapse mortality, and relapse incidence are greatly dependent upon the pre-transplant management. Based on these premises, in this review, we will go through the path of identification of the MF patients suitable for a transplant, by using disease-specific prognostic scores, and the evaluation of eligibility for a transplant, based on performance, comorbidity, and other combined tools. Then, we will focus on the process of donor and conditioning regimens’ choice. The pre-transplant management of splenomegaly and constitutional symptoms, cytopenias, iron overload and transplant timing will be comprehensively discussed. The principal aim of this review is, therefore, to give a practical guidance for managing MF patients who are potential candidates for allo-HCT.
Highlights
Unit of Blood Diseases and Bone Marrow Transplantation, Cell Therapies and Hematology Research Program, Department of Clinical and Experimental Sciences, University of Brescia, ASST Spedali Civili di Brescia, Hematology Division, Department of Oncology, ASST Spedali Civili di Brescia, P.le Spedali Civili 1, 2nd Division of General Surgery, Department of Medical & Surgical Sciences, ASST Spedali Civili di Brescia, Department of Radiology, ASST Spedali Civili di Brescia, P.le Spedali Civili 1, 25123 Brescia, BS, Italy; CREA Laboratory (Centro di Ricerca Emato-Oncologica AIL), ASST Spedali Civili di Brescia, P.le Spedali Civili 1, 25123 Brescia, BS, Italy
Introduction with regard to jurisdictional claims in Allogeneic hematopoietic stem cell transplantation still represents the only curative option for patients with myelofibrosis (MF), a myeloproliferative neoplasm characterized by splenomegaly, constitutional symptoms, anemia, and a natural progression to acute leukemia [1]
Novel and less toxic conditioning platforms, as well as a better HLA donor selection and GVHD and anti-infective therapies, greatly improved the feasibility and safety of allo-HCT. All these advances led to extending the indication for a transplant to a larger number of elderly patients affected by hematological neoplasms, including MF [2]
Summary
Clinical prognostic scoring systems play a pivotal role for guiding selection of MF patient who may benefit from allo-HCT. These data seem to further confirm the validity of ELN/EBMT 2015 indications, where intermediate Such scores have been proven to poorly predict patients’ outcome for patients with secondary myelofibrosis (sMF; post-Polycythemia Vera (PPV-MF) or postEssential Thrombocythemia myelofibrosis (PET-MF)), as they present a better survival compared to primary myelofibrosis [17,18]. Recent updates to ‘MIPSS70-plus version 20 occurred with the recognition of U2AF1Q157 and new sex- and severity-adjusted hemoglobin thresholds [29] These scores incorporate current molecular data and up-to-date WHO 2016 disease classification and can aid decisions regarding allo-HCT. For example, often, a patient with MF may present different IPSS and MIPSS-70 scores with significant difference in OS This kind of discrepancy between risk models may occur in up to 50% of patients with sMF [20], resulting in significative challenges in transplant indication. Unfavorable karyotype: complex karyotype or single or two abnormalities, including +8, −7/7q-, i(17q), −5/5q-, 12p-, inv(3), or 11q23 rearrangement. % HMR mutations according to MIPSS70: ASXL1, SRSF2, EZH2, IDH1, IDH2. $ HMR mutations according to MIPSS70-plus v2.0: ASXL1, SRSF2, EZH2, IDH1, IDH2, and U2AF1Q157. § Very unfavorable karyotype: single/multiple abnormalities of −7, i(17q), inv(3)/3q21, 12p-/12p11.2, 11q-/11q23, or other autosomal trisomies, not including +8/+9 (e.g., +21, +19); Favorable: normal karyotype or sole abnormalities of 13q-, +9, 20q-, chromosome 1 translocation/duplication or sex chromosome abnormality, including -Y; ‘Unfavorable‘: all other abnormalities
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