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Vol 8, No 2 (2017)
Review paper
Published online: 2017-09-06
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Allogeneic hematopoietic stem cell transplantation for treating patients with myelofibrosis

Elżbieta Patkowska, Joanna Góra-Tybor
DOI: 10.5603/Hem.2017.0016
·
Hematologia 2017;8(2):132-143.

open access

Vol 8, No 2 (2017)
REVIEW ARTICLES
Published online: 2017-09-06

Abstract

Within Ph-negative myeloproliferative types of cancer, myelofibrosis is the most frequent indication for performing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Because this is a high-risk procedure, only those patients with a predicted overall survival below 5 years become eligible, as well as those of intermediate-2 or high disease risk; as defined by the IPSS, DIPSS and DIPSS Plus prognostic indices. It has however recently been recognised that patients with a poor prognosis, who belong to the intermediate-1 disease risk group, need to be distinguished and also considered for allo-HSCT. According to ELN/EBMT recommendations from 2015, this group should include patients requiring red blood cell concentrate transfusions who have more than 2% blasts in the circulation and/or an adverse karyotype. When qualifying patients for transplantation, consideration should also be given to adverse molecular risk factors such as the so-called triple negativity (absence of JAK, CALR and MPL mutations) and the presence of ASXL1 mutation. Over recent years, the number of allo-HSCT performed on patients with MF has significantly risen because the number of transplantations using reduced-intensity conditioning regimens has increased, together with those from unrelated donors (cord blood, haploidentical donors). There is a lack of randomised studies comparing the effectiveness of this transplantation procedure according to the types of conditioning and donor selection.

Abstract

Within Ph-negative myeloproliferative types of cancer, myelofibrosis is the most frequent indication for performing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Because this is a high-risk procedure, only those patients with a predicted overall survival below 5 years become eligible, as well as those of intermediate-2 or high disease risk; as defined by the IPSS, DIPSS and DIPSS Plus prognostic indices. It has however recently been recognised that patients with a poor prognosis, who belong to the intermediate-1 disease risk group, need to be distinguished and also considered for allo-HSCT. According to ELN/EBMT recommendations from 2015, this group should include patients requiring red blood cell concentrate transfusions who have more than 2% blasts in the circulation and/or an adverse karyotype. When qualifying patients for transplantation, consideration should also be given to adverse molecular risk factors such as the so-called triple negativity (absence of JAK, CALR and MPL mutations) and the presence of ASXL1 mutation. Over recent years, the number of allo-HSCT performed on patients with MF has significantly risen because the number of transplantations using reduced-intensity conditioning regimens has increased, together with those from unrelated donors (cord blood, haploidentical donors). There is a lack of randomised studies comparing the effectiveness of this transplantation procedure according to the types of conditioning and donor selection.

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Keywords

myelofibrosis, transplantation, allogeneic hematopoietic stem cell transplantation (allo-HSCT), transplantation, reduced-intensity conditioning (RIC), myeloablative conditioning (MAC), ruxolitinib

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Tabela 1 Wyniki przeszczepiania alogenicznych krwiotwórczych komórek macierzystych u chorych na mielofibrozę na podstawie wybranych analiz
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About this article
Title

Allogeneic hematopoietic stem cell transplantation for treating patients with myelofibrosis

Journal

Hematology in Clinical Practice

Issue

Vol 8, No 2 (2017)

Article type

Review paper

Pages

132-143

Published online

2017-09-06

DOI

10.5603/Hem.2017.0016

Bibliographic record

Hematologia 2017;8(2):132-143.

Keywords

myelofibrosis
transplantation
allogeneic hematopoietic stem cell transplantation (allo-HSCT)
transplantation
reduced-intensity conditioning (RIC)
myeloablative conditioning (MAC)
ruxolitinib

Authors

Elżbieta Patkowska
Joanna Góra-Tybor

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