OBJECTIVES: In patients with very severe aplastic anemia (VSAA), neutropenia is prolonged and persistent, resulting in refractory overwhelming infections. Hematopoiesis recovery is urgently needed. METHODS: Six patients with de novo VSAA lacking HLA-identical sibling donors and those who experienced refractory infections underwent haploidentical related donor (HRD) hematopoietic stem cell transplantation (HSCT) as a first-line therapy. The conditioning regimen consisted of busulfan, cyclophosphamide, and rabbit antithymocyte globulin. Culture-expanded allogeneic bone marrow-derived mesenchymal stromal cells were infused on day 0 and day +14. RESULTS: From diagnosis to HSCT, 6 patients experienced a total of 28 episodes of persistent fever, and the median number was 4 (range, 3-7). All cases developed major bacterial infections and invasive pulmonary fungal infection pre-HSCT. The median time from diagnosis to HSCT was 2 months (range, 1-3.5 months). All patients achieved sustained, full donor chimerism, and the median time of myeloid recovery and platelet engraftment was 13 days (range, 9-19 days) and 15.5 days (range, 10-23 days), respectively. One patient died of aGVHD, and 5 patients are alive after a median follow-up of 21 months (range 17-40.5). CONCLUSIONS: Upfront HRD-HSCT may be a safe and promising choice for patients with VSAA in critical situations without suitably matched donors.
OBJECTIVES: In patients with very severe aplastic anemia (VSAA), neutropenia is prolonged and persistent, resulting in refractory overwhelming infections. Hematopoiesis recovery is urgently needed. METHODS: Six patients with de novo VSAA lacking HLA-identical sibling donors and those who experienced refractory infections underwent haploidentical related donor (HRD) hematopoietic stem cell transplantation (HSCT) as a first-line therapy. The conditioning regimen consisted of busulfan, cyclophosphamide, and rabbit antithymocyte globulin. Culture-expanded allogeneic bone marrow-derived mesenchymal stromal cells were infused on day 0 and day +14. RESULTS: From diagnosis to HSCT, 6 patients experienced a total of 28 episodes of persistent fever, and the median number was 4 (range, 3-7). All cases developed major bacterial infections and invasive pulmonary fungal infection pre-HSCT. The median time from diagnosis to HSCT was 2 months (range, 1-3.5 months). All patients achieved sustained, full donor chimerism, and the median time of myeloid recovery and platelet engraftment was 13 days (range, 9-19 days) and 15.5 days (range, 10-23 days), respectively. One patient died of aGVHD, and 5 patients are alive after a median follow-up of 21 months (range 17-40.5). CONCLUSIONS: Upfront HRD-HSCT may be a safe and promising choice for patients with VSAA in critical situations without suitably matched donors.
Authors: Ghada ElGohary; Riad El Fakih; Regis de Latour; Antonio Risitano; Judith Marsh; Hubert Schrezenmeier; Eliane Gluckman; Britta Höchsmann; Filomena Pierri; Constantijn Halkes; Hazzaa Alzahrani; Josu De la Fuente; Simone Cesaro; Ali Alahmari; Syed Osman Ahmed; Jakob Passweg; Carlo Dufour; Andrea Bacigalupo; Mahmoud Aljurf Journal: Bone Marrow Transplant Date: 2020-04-28 Impact factor: 5.483
Authors: Raheel Iftikhar; Parvez Ahmad; Regis de Latour; Carlo Dufour; Antonio Risitano; Naeem Chaudhri; Ali Bazarbachi; Josu De La Fuente; Britta Höchsmann; Syed Osman Ahmed; Usama Gergis; Alaa Elhaddad; Constantijn Halkes; Bassim Albeirouti; Sultan Alotaibi; Austin Kulasekararaj; Hazzaa Alzahrani; Tarek Ben Othman; Simone Cesaro; Ali Alahmari; Rawad Rihani; Salem Alshemmari; Amir Ali Hamidieh; Mohamed-Amine Bekadja; Jakob Passweg; Murtadha Al-Khabori; Walid Rasheed; Andrea Bacigalupo; Qamar-Un-Nisa Chaudhry; Per Ljungman; Judith Marsh; Riad El Fakih; Mahmoud Aljurf Journal: Bone Marrow Transplant Date: 2021-05-19 Impact factor: 5.483