Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel

T. de Witte, D. Bowen, M. Robin, L. Malcovati, D. Niederwieser, I. Yakoub-Agha, G. J. Mufti, P. Fenaux, G. Sanz, R. Martino, E. P. Alessandrino, F. Onida, A. Symeonidis, J. Passweg, G. Kobbe, A. Ganser, U. Platzbecker, J. Finke, M. van Gelder, A. A. van de LoosdrechtP. Ljungman, R. Stauder, L. Volin, H. J. Deeg, C. Cutler, W. Saber, R. Champlin, S. Giralt, C. Anasetti, N. Kroger

Research output: Contribution to journalArticle

Abstract

An international expert panel, active within the European Society for Blood and Marrow Transplantation, European LeukemiaNet, Blood and Marrow Transplant Clinical Trial Group, and the International Myelodysplastic Syndromes Foundation developed recommendations for allogeneic hematopoietic stem cell transplantation (HSCT) in myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Disease risks scored according to the revised International Prognostic Scoring System (IPSS-R) and presence of comorbidity graded according to the HCT Comorbidity Index (HCT-CI) were recognized as relevant clinical variables for HSCT eligibility. Fit patients with higher-risk IPSS-R and those with lower-risk IPSS-R with poor-risk genetic features, profound cytopenias, and high transfusion burden are candidates for HSCT. Patients with a very high MDS transplantation risk score, based on combination of advanced age, high HCT-CI, very poor-risk cytogenetic and molecular features, and high IPSS-R score have a low chance of cure with standard HSCT and consideration should be given to treating these patients in investigational studies. Cytoreductive therapy prior to HSCT is advised for patients with >/=10% bone marrow myeloblasts. Evidence from prospective randomized clinical trials does not provide support for specific recommendations on the optimal high intensity conditioning regimen. For patients with contraindications to high-intensity preparative regimens, reduced intensity conditioning should be considered. Optimal timing of HSCT requires careful evaluation of the available effective nontransplant strategies. Prophylactic donor lymphocyte infusion (DLI) strategies are recommended in patients at high risk of relapse after HSCT. Immune modulation by DLI strategies or second HSCT is advised if relapse occurs beyond 6 months after HSCT.
Original languageEnglish
Pages (from-to)1753-1762
Number of pages10
JournalBlood
Volume129
Issue number13
DOIs
Publication statusPublished - Mar 30 2017

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Leukemia, Myelomonocytic, Chronic
Hematopoietic Stem Cell Transplantation
Myelodysplastic Syndromes
Stem cells
Comorbidity
Lymphocytes
Bone Marrow
Blood
Transplantation
Tissue Donors
Transplantation (surgical)
Recurrence
Granulocyte Precursor Cells
Transplants
Cytogenetics
Bone
Randomized Controlled Trials
Modulation
Clinical Trials

Keywords

  • Hematopoietic Stem Cell Transplantation/methods
  • Humans
  • Leukemia, Myelomonocytic, Chronic/therapy
  • Myelodysplastic Syndromes/therapy
  • Practice Guidelines as Topic
  • Risk Factors
  • Transplantation Conditioning
  • Transplantation, Homologous

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Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. / Witte, T. de; Bowen, D.; Robin, M.; Malcovati, L.; Niederwieser, D.; Yakoub-Agha, I.; Mufti, G. J.; Fenaux, P.; Sanz, G.; Martino, R.; Alessandrino, E. P.; Onida, F.; Symeonidis, A.; Passweg, J.; Kobbe, G.; Ganser, A.; Platzbecker, U.; Finke, J.; Gelder, M. van; Loosdrecht, A. A. van de; Ljungman, P.; Stauder, R.; Volin, L.; Deeg, H. J.; Cutler, C.; Saber, W.; Champlin, R.; Giralt, S.; Anasetti, C.; Kroger, N.

In: Blood, Vol. 129, No. 13, 30.03.2017, p. 1753-1762.

Research output: Contribution to journalArticle

Witte, TD, Bowen, D, Robin, M, Malcovati, L, Niederwieser, D, Yakoub-Agha, I, Mufti, GJ, Fenaux, P, Sanz, G, Martino, R, Alessandrino, EP, Onida, F, Symeonidis, A, Passweg, J, Kobbe, G, Ganser, A, Platzbecker, U, Finke, J, Gelder, MV, Loosdrecht, AAVD, Ljungman, P, Stauder, R, Volin, L, Deeg, HJ, Cutler, C, Saber, W, Champlin, R, Giralt, S, Anasetti, C & Kroger, N 2017, 'Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel', Blood, vol. 129, no. 13, pp. 1753-1762. https://doi.org/10.1182/blood-2016-06-724500 [doi]
Witte, T. de ; Bowen, D. ; Robin, M. ; Malcovati, L. ; Niederwieser, D. ; Yakoub-Agha, I. ; Mufti, G. J. ; Fenaux, P. ; Sanz, G. ; Martino, R. ; Alessandrino, E. P. ; Onida, F. ; Symeonidis, A. ; Passweg, J. ; Kobbe, G. ; Ganser, A. ; Platzbecker, U. ; Finke, J. ; Gelder, M. van ; Loosdrecht, A. A. van de ; Ljungman, P. ; Stauder, R. ; Volin, L. ; Deeg, H. J. ; Cutler, C. ; Saber, W. ; Champlin, R. ; Giralt, S. ; Anasetti, C. ; Kroger, N. / Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. In: Blood. 2017 ; Vol. 129, No. 13. pp. 1753-1762.
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T1 - Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel

AU - Witte, T. de

AU - Bowen, D.

AU - Robin, M.

AU - Malcovati, L.

AU - Niederwieser, D.

AU - Yakoub-Agha, I.

AU - Mufti, G. J.

AU - Fenaux, P.

AU - Sanz, G.

AU - Martino, R.

AU - Alessandrino, E. P.

AU - Onida, F.

AU - Symeonidis, A.

AU - Passweg, J.

AU - Kobbe, G.

AU - Ganser, A.

AU - Platzbecker, U.

AU - Finke, J.

AU - Gelder, M. van

AU - Loosdrecht, A. A. van de

AU - Ljungman, P.

AU - Stauder, R.

AU - Volin, L.

AU - Deeg, H. J.

AU - Cutler, C.

AU - Saber, W.

AU - Champlin, R.

AU - Giralt, S.

AU - Anasetti, C.

AU - Kroger, N.

N1 - LR: 20180112; CI: (c) 2017; GR: P30 CA008748/CA/NCI NIH HHS/United States; JID: 7603509; PMCR: 2018/03/30 00:00; 2016/06/25 00:00 [received]; 2017/01/04 00:00 [accepted]; 2018/03/30 00:00 [pmc-release]; 2017/01/18 06:00 [pubmed]; 2017/08/24 06:00 [medline]; 2017/01/19 06:00 [entrez]; ppublish

PY - 2017/3/30

Y1 - 2017/3/30

N2 - An international expert panel, active within the European Society for Blood and Marrow Transplantation, European LeukemiaNet, Blood and Marrow Transplant Clinical Trial Group, and the International Myelodysplastic Syndromes Foundation developed recommendations for allogeneic hematopoietic stem cell transplantation (HSCT) in myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Disease risks scored according to the revised International Prognostic Scoring System (IPSS-R) and presence of comorbidity graded according to the HCT Comorbidity Index (HCT-CI) were recognized as relevant clinical variables for HSCT eligibility. Fit patients with higher-risk IPSS-R and those with lower-risk IPSS-R with poor-risk genetic features, profound cytopenias, and high transfusion burden are candidates for HSCT. Patients with a very high MDS transplantation risk score, based on combination of advanced age, high HCT-CI, very poor-risk cytogenetic and molecular features, and high IPSS-R score have a low chance of cure with standard HSCT and consideration should be given to treating these patients in investigational studies. Cytoreductive therapy prior to HSCT is advised for patients with >/=10% bone marrow myeloblasts. Evidence from prospective randomized clinical trials does not provide support for specific recommendations on the optimal high intensity conditioning regimen. For patients with contraindications to high-intensity preparative regimens, reduced intensity conditioning should be considered. Optimal timing of HSCT requires careful evaluation of the available effective nontransplant strategies. Prophylactic donor lymphocyte infusion (DLI) strategies are recommended in patients at high risk of relapse after HSCT. Immune modulation by DLI strategies or second HSCT is advised if relapse occurs beyond 6 months after HSCT.

AB - An international expert panel, active within the European Society for Blood and Marrow Transplantation, European LeukemiaNet, Blood and Marrow Transplant Clinical Trial Group, and the International Myelodysplastic Syndromes Foundation developed recommendations for allogeneic hematopoietic stem cell transplantation (HSCT) in myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Disease risks scored according to the revised International Prognostic Scoring System (IPSS-R) and presence of comorbidity graded according to the HCT Comorbidity Index (HCT-CI) were recognized as relevant clinical variables for HSCT eligibility. Fit patients with higher-risk IPSS-R and those with lower-risk IPSS-R with poor-risk genetic features, profound cytopenias, and high transfusion burden are candidates for HSCT. Patients with a very high MDS transplantation risk score, based on combination of advanced age, high HCT-CI, very poor-risk cytogenetic and molecular features, and high IPSS-R score have a low chance of cure with standard HSCT and consideration should be given to treating these patients in investigational studies. Cytoreductive therapy prior to HSCT is advised for patients with >/=10% bone marrow myeloblasts. Evidence from prospective randomized clinical trials does not provide support for specific recommendations on the optimal high intensity conditioning regimen. For patients with contraindications to high-intensity preparative regimens, reduced intensity conditioning should be considered. Optimal timing of HSCT requires careful evaluation of the available effective nontransplant strategies. Prophylactic donor lymphocyte infusion (DLI) strategies are recommended in patients at high risk of relapse after HSCT. Immune modulation by DLI strategies or second HSCT is advised if relapse occurs beyond 6 months after HSCT.

KW - Hematopoietic Stem Cell Transplantation/methods

KW - Humans

KW - Leukemia, Myelomonocytic, Chronic/therapy

KW - Myelodysplastic Syndromes/therapy

KW - Practice Guidelines as Topic

KW - Risk Factors

KW - Transplantation Conditioning

KW - Transplantation, Homologous

U2 - 10.1182/blood-2016-06-724500 [doi]

DO - 10.1182/blood-2016-06-724500 [doi]

M3 - Article

VL - 129

SP - 1753

EP - 1762

JO - Blood

JF - Blood

SN - 0006-4971

IS - 13

ER -