George E Georges1, Rainer Storb. 1. aClinical Research Division, Fred Hutchinson Cancer Research Center bDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Abstract
PURPOSE OF REVIEW: There has been a steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), because of progress in optimization of the conditioning regimens, donor hematopoietic cell source, and supportive care. Here, we review recently published data that highlight the improvements and current issues in the treatment of SAA. RECENT FINDINGS: Approximately one-third of aplastic anemia patients treated with immune suppressive therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using posttransplant cyclophosphamide conditioning regimens is now a reasonable second-line treatment for patients who failed IST. SUMMARY: Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors.
PURPOSE OF REVIEW: There has been a steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), because of progress in optimization of the conditioning regimens, donor hematopoietic cell source, and supportive care. Here, we review recently published data that highlight the improvements and current issues in the treatment of SAA. RECENT FINDINGS: Approximately one-third of aplastic anemiapatients treated with immune suppressive therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using posttransplant cyclophosphamide conditioning regimens is now a reasonable second-line treatment for patients who failed IST. SUMMARY: Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors.
Authors: H Joachim Deeg; Margaret O'Donnell; Jakub Tolar; Rajni Agarwal; Richard E Harris; Stephen A Feig; Mary C Territo; Robert H Collins; Peter A McSweeney; Edward A Copelan; Shakila P Khan; Ann Woolfrey; Barry Storer Journal: Blood Date: 2006-05-09 Impact factor: 22.113
Authors: I Esteves; C Bonfim; R Pasquini; V Funke; N F Pereira; V Rocha; Y Novis; C Arrais; V Colturato; M P de Souza; M Torres; J F Fernandes; F R Kerbauy; A A F Ribeiro; F P S Santos; N Hamerschlak Journal: Bone Marrow Transplant Date: 2015-03-02 Impact factor: 5.483
Authors: Andrea Bacigalupo; Gerard Socié; Rose Marie Hamladji; Mahmoud Aljurf; Alexei Maschan; Slawomira Kyrcz-Krzemien; Alicja Cybicka; Henrik Sengelov; Ali Unal; Dietrich Beelen; Anna Locasciulli; Carlo Dufour; Jakob R Passweg; Rosi Oneto; Alessio Signori; Judith C W Marsh Journal: Haematologica Date: 2015-01-23 Impact factor: 9.941
Authors: Carlo Dufour; Paul Veys; Elisa Carraro; Neha Bhatnagar; Marta Pillon; Rob Wynn; Brenda Gibson; Ajay J Vora; Colin G Steward; Anna M Ewins; Rachael E Hough; Josu de la Fuente; Mark Velangi; Persis J Amrolia; Roderick Skinner; Andrea Bacigalupo; Antonio M Risitano; Gerard Socie; Regis Peffault de Latour; Jakob Passweg; Alicia Rovo; André Tichelli; Hubert Schrezenmeier; Britta Hochsmann; Peter Bader; Anja van Biezen; Mahmoud D Aljurf; Austin Kulasekararaj; Judith C Marsh; Sujith Samarasinghe Journal: Br J Haematol Date: 2015-07-28 Impact factor: 6.998
Authors: J C Marsh; R M Pearce; M B C Koh; Z Lim; A Pagliuca; G J Mufti; J Perry; J A Snowden; A J Vora; R T Wynn; N Russell; B Gibson; M Gilleece; D Milligan; P Veys; S Samarasinghe; M McMullin; K Kirkland; G Cook Journal: Bone Marrow Transplant Date: 2013-08-05 Impact factor: 5.483