Impact of drug development on the use of stem cell transplantation: a report by the European Society for Blood and Marrow Transplantation (EBMT)

Jakob Passweg, H. Baldomero, P. Bader, Chiara Bonini, S. Cesaro, P. Dreger, R. F. Duarte, Carlo Dufour, Jürgen H E Kuball, Dominique Farge-Bancel, Andrew Gennery, Nicolaus Kröger, Francesco Lanza, Arnon Nagler, Anna Sureda, Mohamad Mohty

Research output: Contribution to journalArticle

Abstract

Hematopoietic stem cell transplantation (HSCT) is used with increasing frequency in Europe with 40 000 transplants reported in 2014. Transplant-related mortality remains high in allogeneic HSCT (10–20%); high-dose chemotherapy is toxic and demanding for patients. Drug development is accelerating and with limited toxicity of some targeted drugs may replace HSCT, whereas others may function as a ‘bridge to transplant’. We analyzed HSCT reported to the activity survey for selected diseases in which major advances in drug development have been made. Tyrosine kinase inhibitors markedly changed the number of allogeneic HSCT in early CML. In myelodysplastic syndromes, hypomethylating agents show no effect on HSCT activity and Janus kinase inhibitors for myeloproliferative neoplasm appear to have only a temporary effect. For CLL autologous HSCT decreased after publication of trials showing improved PFS but no overall survival advantage and allogeneic rates are dropping after the introduction of Bruton kinase and PI3K Inhibitors. Whether these are ‘game changers’ as was imatinib for CML requires additional follow-up. For myeloma, proteasome inhibitors and new immunomodulatory drugs do not appear to impact transplant rates. Drug development data show different effects on HSCT use; highly effective drugs may replace HSCT, whereas other drugs may improve the patient’s condition to allow for HSCT.Bone Marrow Transplantation advance online publication, 7 November 2016; doi:10.1038/bmt.2016.258.

Original languageEnglish
JournalBone Marrow Transplantation
DOIs
Publication statusAccepted/In press - Nov 7 2016

Fingerprint

Hematopoietic Stem Cell Transplantation
Stem Cell Transplantation
Transplantation
Bone Marrow
Pharmaceutical Preparations
Transplants
Publications
Janus Kinases
Proteasome Inhibitors
Poisons
Myelodysplastic Syndromes
Bone Marrow Transplantation
Phosphatidylinositol 3-Kinases
Protein-Tyrosine Kinases
Phosphotransferases
Drug Therapy
Survival
Mortality

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Impact of drug development on the use of stem cell transplantation : a report by the European Society for Blood and Marrow Transplantation (EBMT). / Passweg, Jakob; Baldomero, H.; Bader, P.; Bonini, Chiara; Cesaro, S.; Dreger, P.; Duarte, R. F.; Dufour, Carlo; Kuball, Jürgen H E; Farge-Bancel, Dominique; Gennery, Andrew; Kröger, Nicolaus; Lanza, Francesco; Nagler, Arnon; Sureda, Anna; Mohty, Mohamad.

In: Bone Marrow Transplantation, 07.11.2016.

Research output: Contribution to journalArticle

Passweg, J, Baldomero, H, Bader, P, Bonini, C, Cesaro, S, Dreger, P, Duarte, RF, Dufour, C, Kuball, JHE, Farge-Bancel, D, Gennery, A, Kröger, N, Lanza, F, Nagler, A, Sureda, A & Mohty, M 2016, 'Impact of drug development on the use of stem cell transplantation: a report by the European Society for Blood and Marrow Transplantation (EBMT)', Bone Marrow Transplantation. https://doi.org/10.1038/bmt.2016.258
Passweg, Jakob ; Baldomero, H. ; Bader, P. ; Bonini, Chiara ; Cesaro, S. ; Dreger, P. ; Duarte, R. F. ; Dufour, Carlo ; Kuball, Jürgen H E ; Farge-Bancel, Dominique ; Gennery, Andrew ; Kröger, Nicolaus ; Lanza, Francesco ; Nagler, Arnon ; Sureda, Anna ; Mohty, Mohamad. / Impact of drug development on the use of stem cell transplantation : a report by the European Society for Blood and Marrow Transplantation (EBMT). In: Bone Marrow Transplantation. 2016.
@article{f64af2f0fb6e43248218f6119389634f,
title = "Impact of drug development on the use of stem cell transplantation: a report by the European Society for Blood and Marrow Transplantation (EBMT)",
abstract = "Hematopoietic stem cell transplantation (HSCT) is used with increasing frequency in Europe with 40 000 transplants reported in 2014. Transplant-related mortality remains high in allogeneic HSCT (10–20{\%}); high-dose chemotherapy is toxic and demanding for patients. Drug development is accelerating and with limited toxicity of some targeted drugs may replace HSCT, whereas others may function as a ‘bridge to transplant’. We analyzed HSCT reported to the activity survey for selected diseases in which major advances in drug development have been made. Tyrosine kinase inhibitors markedly changed the number of allogeneic HSCT in early CML. In myelodysplastic syndromes, hypomethylating agents show no effect on HSCT activity and Janus kinase inhibitors for myeloproliferative neoplasm appear to have only a temporary effect. For CLL autologous HSCT decreased after publication of trials showing improved PFS but no overall survival advantage and allogeneic rates are dropping after the introduction of Bruton kinase and PI3K Inhibitors. Whether these are ‘game changers’ as was imatinib for CML requires additional follow-up. For myeloma, proteasome inhibitors and new immunomodulatory drugs do not appear to impact transplant rates. Drug development data show different effects on HSCT use; highly effective drugs may replace HSCT, whereas other drugs may improve the patient’s condition to allow for HSCT.Bone Marrow Transplantation advance online publication, 7 November 2016; doi:10.1038/bmt.2016.258.",
author = "Jakob Passweg and H. Baldomero and P. Bader and Chiara Bonini and S. Cesaro and P. Dreger and Duarte, {R. F.} and Carlo Dufour and Kuball, {J{\"u}rgen H E} and Dominique Farge-Bancel and Andrew Gennery and Nicolaus Kr{\"o}ger and Francesco Lanza and Arnon Nagler and Anna Sureda and Mohamad Mohty",
year = "2016",
month = "11",
day = "7",
doi = "10.1038/bmt.2016.258",
language = "English",
journal = "Bone Marrow Transplantation",
issn = "0268-3369",
publisher = "Nature Publishing Group",

}

TY - JOUR

T1 - Impact of drug development on the use of stem cell transplantation

T2 - a report by the European Society for Blood and Marrow Transplantation (EBMT)

AU - Passweg, Jakob

AU - Baldomero, H.

AU - Bader, P.

AU - Bonini, Chiara

AU - Cesaro, S.

AU - Dreger, P.

AU - Duarte, R. F.

AU - Dufour, Carlo

AU - Kuball, Jürgen H E

AU - Farge-Bancel, Dominique

AU - Gennery, Andrew

AU - Kröger, Nicolaus

AU - Lanza, Francesco

AU - Nagler, Arnon

AU - Sureda, Anna

AU - Mohty, Mohamad

PY - 2016/11/7

Y1 - 2016/11/7

N2 - Hematopoietic stem cell transplantation (HSCT) is used with increasing frequency in Europe with 40 000 transplants reported in 2014. Transplant-related mortality remains high in allogeneic HSCT (10–20%); high-dose chemotherapy is toxic and demanding for patients. Drug development is accelerating and with limited toxicity of some targeted drugs may replace HSCT, whereas others may function as a ‘bridge to transplant’. We analyzed HSCT reported to the activity survey for selected diseases in which major advances in drug development have been made. Tyrosine kinase inhibitors markedly changed the number of allogeneic HSCT in early CML. In myelodysplastic syndromes, hypomethylating agents show no effect on HSCT activity and Janus kinase inhibitors for myeloproliferative neoplasm appear to have only a temporary effect. For CLL autologous HSCT decreased after publication of trials showing improved PFS but no overall survival advantage and allogeneic rates are dropping after the introduction of Bruton kinase and PI3K Inhibitors. Whether these are ‘game changers’ as was imatinib for CML requires additional follow-up. For myeloma, proteasome inhibitors and new immunomodulatory drugs do not appear to impact transplant rates. Drug development data show different effects on HSCT use; highly effective drugs may replace HSCT, whereas other drugs may improve the patient’s condition to allow for HSCT.Bone Marrow Transplantation advance online publication, 7 November 2016; doi:10.1038/bmt.2016.258.

AB - Hematopoietic stem cell transplantation (HSCT) is used with increasing frequency in Europe with 40 000 transplants reported in 2014. Transplant-related mortality remains high in allogeneic HSCT (10–20%); high-dose chemotherapy is toxic and demanding for patients. Drug development is accelerating and with limited toxicity of some targeted drugs may replace HSCT, whereas others may function as a ‘bridge to transplant’. We analyzed HSCT reported to the activity survey for selected diseases in which major advances in drug development have been made. Tyrosine kinase inhibitors markedly changed the number of allogeneic HSCT in early CML. In myelodysplastic syndromes, hypomethylating agents show no effect on HSCT activity and Janus kinase inhibitors for myeloproliferative neoplasm appear to have only a temporary effect. For CLL autologous HSCT decreased after publication of trials showing improved PFS but no overall survival advantage and allogeneic rates are dropping after the introduction of Bruton kinase and PI3K Inhibitors. Whether these are ‘game changers’ as was imatinib for CML requires additional follow-up. For myeloma, proteasome inhibitors and new immunomodulatory drugs do not appear to impact transplant rates. Drug development data show different effects on HSCT use; highly effective drugs may replace HSCT, whereas other drugs may improve the patient’s condition to allow for HSCT.Bone Marrow Transplantation advance online publication, 7 November 2016; doi:10.1038/bmt.2016.258.

UR - http://www.scopus.com/inward/record.url?scp=84994575954&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84994575954&partnerID=8YFLogxK

U2 - 10.1038/bmt.2016.258

DO - 10.1038/bmt.2016.258

M3 - Article

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

SN - 0268-3369

ER -