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,Helen Baldomero,Peter Bader,Chiara Bonini,Simone Cesaro,Peter Dreger,R. F. Duarte,Carlo Dufour,Jürgen Kuball,D Farge-Bancel,Andrew R. Gennery,Nicolaus Kröger,Francesco Lanza,Arnon Nagler,Anna Sureda,Mohamad Mohty +15 more
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TLDR
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.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.read more
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Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report
Jakob Passweg,Helen Baldomero,Peter Bader,Chiara Bonini,R. F. Duarte,Carlo Dufour,Andrew R. Gennery,Kröger N,Jürgen Kuball,Francesco Lanza,Silvia Montoto,Arnon Nagler,John A. Snowden,Jan Styczyński,Mohamad Mohty +14 more
TL;DR: Use of haploidentical donors for allogeneic HSCT continues to grow: 2012 in 2015, a 291% increase since 2005, and growth is seen for all diseases.
Journal ArticleDOI
Is the use of unrelated donor transplantation leveling off in Europe? The 2016 European Society for Blood and Marrow Transplant activity survey report.
Jakob Passweg,Helen Baldomero,Peter Bader,Grzegorz W. Basak,Chiara Bonini,Rafael F. Duarte,Carlo Dufour,Nicolaus Kröger,Jürgen Kuball,Arjan C. Lankester,Silvia Montoto,Arnon Nagler,John A. Snowden,Jan Styczyński,Mohamad Mohty +14 more
TL;DR: There was a remarkable leveling off in the use of unrelated donor HCT being replaced by haploidentical HCT, and continued growth in allogeneic HCT for marrow failure, AML, and MPN was seen, whereas MDS appears stable.
Journal ArticleDOI
Hematopoietic stem cell transplantation in its 60s: A platform for cellular therapies.
Christian Chabannon,Jürgen Kuball,Attilio Bondanza,Francesco Dazzi,Paolo Pedrazzoli,Antoine Toubert,Annalisa Ruggeri,Katharina Fleischhauer,Chiara Bonini +8 more
TL;DR: Improved understanding of underlying biological processes resulted in the design of innovative therapies engineered from defined cell populations and testing of these therapies as addition or substitution at virtually every step of the procedure.
Journal ArticleDOI
Who Is the Patient at Risk of CMV Recurrence: A Review of the Current Scientific Evidence with a Focus on Hematopoietic Cell Transplantation
TL;DR: Data is presented on the incidence of CMV recurrence in groups of immunocompromised patients, including allogeneic hematopoietic stem cell transplantation (HSCT) patients and other groups of patients, based on a summary of reported data.
Journal ArticleDOI
How and when I do allogeneic transplant in CLL
TL;DR: The different treatment options available for the treatment of high-risk CLL and how allo-SCT fits into the treatment algorithm in the era of novel agents are discussed.
References
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Journal ArticleDOI
Hematopoietic Stem-Cell Transplantation
TL;DR: Hematopoietic stem-cell transplantation was first conceived more than 50 years ago, but problems associated with transplanting a nonsolid organ and modulating the immune response had to be solved before the procedure could be used clinically as mentioned in this paper.
Journal ArticleDOI
Reduced mortality after allogeneic hematopoietic-cell transplantation.
Ted Gooley,Jason W. Chien,Jason W. Chien,Steven A. Pergam,Steven A. Pergam,Sangeeta Hingorani,Sangeeta Hingorani,Mohamed L. Sorror,Mohamed L. Sorror,Michael Boeckh,Michael Boeckh,Paul J. Martin,Paul J. Martin,Brenda M. Sandmaier,Brenda M. Sandmaier,Kieren A. Marr,Kieren A. Marr,Frederick R. Appelbaum,Frederick R. Appelbaum,Rainer Storb,Rainer Storb,George B. McDonald,George B. McDonald +22 more
TL;DR: A substantial reduction in the hazard of death related to allogeneic hematopoietic-cell transplantation, as well as increased long-term survival, over the past decade is found.
Journal ArticleDOI
High-dose chemotherapy with hematopoietic rescue as primary treatment for metastatic breast cancer: a randomized trial.
TL;DR: HDR-CNV appears to be a promising schedule that results in a significant proportion of CRs and increased survival in patients with metastatic breast cancer.
Journal ArticleDOI
Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia
Matthew J. Olnes,Phillip Scheinberg,Katherine R. Calvo,Ronan Desmond,Yong Tang,Bogdan Dumitriu,Ankur R. Parikh,Susan Soto,Angelique Biancotto,Xingmin Feng,Jay N. Lozier,Colin O. Wu,Neal S. Young,Cynthia E. Dunbar +13 more
TL;DR: Treatment with eltrombopag was associated with multilineage clinical responses in some patients with refractory severe aplastic anemia and may improve blood counts.
Journal ArticleDOI
High-dose combination alkylating agents with bone marrow support as initial treatment for metastatic breast cancer.
William P. Peters,Elizabeth J. Shpall,Roy B. Jones,G. A. Olsen,Robert C. Bast,Jon P. Gockerman,Joseph O. Moore +6 more
TL;DR: It is indicated that a single treatment with intensive combination alkylating agents with bone marrow support can produce more rapid and frequent complete responses than conventional chemotherapy when used as initial chemotherapy for metastatic breast cancer, although median disease-free and overall survival is not improved.
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