Impact of drug development on the use of stem cell transplantation: a report by the European Society for Blood and Marrow Transplantation (EBMT).
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.
EBMT Activity Survey Office, Division of Hematology, Department of Medicine, University Hospital, Basel, Switzerland.
J R
JR
Passweg
EBMT Activity Survey Office, Division of Hematology, Department of Medicine, University Hospital, Basel, Switzerland.
H
H
Baldomero
Universitätsklinikum Frankfurt, Goethe-Universität, Frankfurt am Main, Germany.
P
P
Bader
Università Vita-Salute San Raffaele, Milan, Italy.
C
C
Bonini
Pediatric Haematology and Oncology, Policlinico GB Rossi, Verona, Italy.
S
S
Cesaro
Medizinische Klinik V, University of Heidelberg, Heidelberg, Germany.
P
P
Dreger
Hematology Department, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain.
R F
RF
Duarte
Hematology Unit, G Gaslini Children's Institute, Genova, Italy.
C
C
Dufour
Department of Hematology, University Medical Centre, Utrecht, The Netherlands.
J
J
Kuball
Service de Médecine Interne, Maladies auto-immunes et pathologie vasculaire, Hôpital St Louis, Paris, France.
D
D
Farge-Bancel
Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK.
A
A
Gennery
University Hospital Eppendorf, Hamburg, Germany.
N
N
Kröger
Hematology and BMT Unit, University Hospital of Ravenna, Ravenna, Italy.
F
F
Lanza
Chaim Sheba Medical Center, Tel-Hashomer, Israel.
A
A
Nagler
Hematology Department, Institut Català d'Oncologia-Hospital Duran I Reynals, Barcelona, Spain.
A
A
Sureda
Hospital Saint Antoine, Paris, France.
M
M
Mohty
D064591:Allografts; D000970:Antineoplastic Agents / Q000008:administration & dosage*; D055808:Drug Discovery; D005060:Europe; D005260:Female; D019337:Hematologic Neoplasms / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D012955:Societies, Medical
D000970:Antineoplastic Agents