scholarly journals Impact of HLA Mismatch Direction on the Outcome of Unrelated Bone Marrow Transplantation: A Retrospective Analysis from the Japan Society for Hematopoietic Cell Transplantation

2015 ◽  
Vol 21 (2) ◽  
pp. 305-311 ◽  
Author(s):  
Junya Kanda ◽  
Tatsuo Ichinohe ◽  
Shigeo Fuji ◽  
Yoshinobu Maeda ◽  
Kazuteru Ohashi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Retrospective Analysis ◽  
Cell Transplantation ◽  
Marrow Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Japan Society ◽  
Unrelated Bone Marrow Transplantation

Successful Unrelated Bone Marrow Transplantation for Three patients with Amegakaryocytic Thrombocytopenia with Radio-Ulnar Synostosis (CTRUS) in Japan

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4510-4510
Author(s):  
Yoshitoshi Ohtsuka
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Bone Marrow Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Marrow Transplantation ◽  
Hematopoietic Stem ◽  
Exon 2 ◽  
Unrelated Bone Marrow Transplantation ◽  
Reduced Intensity

Abstract 4510 Objectives: Amegakaryocytic thrombocytopenia with radio-ulnar synostosis (CTRUS) is a rare inherited bone marrow failure syndrome that has the potential to progress to pancytopenia that behaves like TAR.The diagnosis of CTRUS is made later when pronation-supination of the forearm is discovered to be restricted. Hox 11a was reported to be abnormal in the initial cases but at least 1 other case did not have this abnormality. Hematopoietic stem-cell transplantation (HSCT) is presently the only curative treatment approach. We used a reduced intensity transplantation regimen in CTRUS patient with aplastic anemia.We reported two cases of CTRUS who underwent successful unrelated bone marrow transplantation. Methods: Conditioning regimen consisted of Flu, ATG, CY, and TLI (300cGy). For GVHD prophylaxis, all received FK506 and short course MTX. The number of transplanted nucleated cells were 6.35, 6.8, 5.8 ×108/kg. All patients underwent an alternative donor SCT. The each age at transplant for patients were 8, 18,11 months. Results: The patients had rapid and durable engraftment at day +13, +16, +23 with minimal complications.No regimen related toxicity were observed, and no viral re-activation (CMV, EBV, VZV) were seen. Each patients developed acute GVHD involving skin only (grade I - II).One of three patients developed chronic GVHD (skin only limited type). All patients are alive and transfusion independent with each follow-up time of 61, 56, 33 months. Conclusions: Allo-BMT from an unrelated donor is a curative and suitable approach for patients with amegakaryocytic thrombocytopenia with radio-ulnar synostosis (CTRUS), reduced intensity conditioning might be a feasible approach to stem-cell transplantation in patients who do not have a sibling donor. In our three cases, the mutation in exon 2 of HOXA11 gene was not found, which may suggest that these cases are possibly a subtype of this syndrome.Careful clinical course watching is required if another complications or the disease relapse will occur again. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Transfusion Support in Patients with Sickle Cell Anemia Undergoing Haploidentical Hematopoietic Cell Transplantation

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3686-3686
Author(s):  
Dilan A Patel ◽  
Deva Sharma ◽  
Jennifer Andrews ◽  
Adetola A. Kassim
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Marrow Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Marrow Transplant ◽  
Cell Disease

Introduction: Hematopoietic cell transplantation (HCT) is the only curative therapy for sickle cell disease (SCD), though is not widely utilized due to inadequate donor availability. Our center has previously reported improved outcomes with a reduced intensity, haploidentical bone marrow transplant (haplo-BMT) with post-transplant Cytoxan and thiotepa (de la Fuente et al, Biol Blood Marrow Transplant 2019). All patients treated with this protocol require red blood cell (RBC) support for anemia. Alloimmunization to minor RBC antigens can be a barrier to therapies including HCT, especially in those patients with multiple antibodies or those requiring rare RBC units. We report on our experience thus far in seven patients with SCA on this protocol. Materials and Methods: After IRB approval, we report in a cohort of 10 patients who completed haplo-BMT with our ongoing platform (ClinicalTrials.gov Identifier: NCT01850108). Phenotypic matching of RBCs beyond ABO and Rh(D) is necessary since patients with SCD commonly produce antibodies against minor RBC antigens, which can result in transfusion reactions such as delayed hemolysis and hyperhemolysis. Our institutional practice is to obtain minor RBC phenotyping on both the patient and their donor prior to transplant. The blood bank provides RBCs that are phenotypically matched for Rh and Kell antigens regardless of antibody status. If a patient has a history of an antibody or a positive screen, the blood bank provides extended phenotypic matching to include Fya, Fyb, Jka, Jkb, and MNS if possible. Outcomes data were analyzed using the CIBMTR database and EMR. Results: A total of 10 patients underwent haplo-BMT, 7 for sickle cell anemia and 3 for beta thalassemia major. Two patients had alloantibodies prior to haplo-BMT, both of whom had SCA. Three patients were ABO identical with their donor; five patients received minor ABO incompatible transplants, and two received major ABO incompatible transplants. Indications for transplant included transfusion dependence (5 patients), cerebrovascular accidents (4 patients), recurrent pain crises (1 patient), visual impairment (1 patient), and seizures (1 patient). The median recipient age at transplant was 17.8 years for the 8 males and 2 females included. Median donor age was 37.5 years, including 4 males and 6 females, with 7 parental donors and 3 sibling donors. No grade 1 acute graft-versus-host disease (GvHD) was noted, though 30% (3/10) had grades II-IV disease. Limited chronic GvHD occurred in 20% (2/10) patients, without any cases of extensive disease. A median of 3.5 RBC (range 0 - 14) transfusions and a median of 10 (range 1 - 24) platelet transfusions were required from date of transplant until neutrophil engraftment. None of the patients had delayed erythroid engraftment. Conclusion: We report successful haploidentical HCT in 7 patients with sickle cell anemia. Providing appropriate phenotypically matched RBCs is especially important in the era of emerging cures for sickle cell anemia, including gene therapy and haploidentical HCT. Alloimmunization is an understudied barrier to HSCT and must be carefully assessed when planning for HCT or other curative therapies required RBC transfusion support. 1. de la Fuente J, Dhedin N, Koyama T, et al. Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Learning Collaborative. Biol Blood Marrow Transplant 2019;25:1197-1209. 2. Bolanos-Meade J, Fuchs EJ, Luznik L, et al. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood 2012;120:4285-91. Disclosures No relevant conflicts of interest to declare.

scholarly journals Impact of Different Doses of Fludarabine in Fludarabine-Based Conditioning Regimen for Unrelated Bone Marrow Transplantation

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3258-3258
Author(s):  
Kodai Kuriyama ◽  
Shigeo Fuji ◽  
Noriko Doki ◽  
Yuta Katayama ◽  
Souichi Shiratori ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Cell Transplantation ◽  
Myeloid Leukemia ◽  
Low Dose ◽  
Hematopoietic Cell Transplantation ◽  
Conditioning Regimen ◽  
Hematopoietic Cell ◽  
High Dose ◽  
The Impact

Background: The purine analog fludarabine (Flu) is recognized as an agent having strong immunosuppressive effects and plays a central role in reduced-intensity conditioning (RIC) and myeloablative reduced-toxicity conditioning (RTC) regimens because of limited nonhematologic toxicities. Combinations of Flu and busulfan (Bu) (FB group) or melphalan (Mel) (FM group) are the commonly used as RIC and RTC regimens. Flu inhibits lymphocyte proliferation, induces apoptosis of hematologic malignant cells and has a synergistic tumor-killing effect with alkylating agents such as Bu and Mel. Few reports assess the impact of different dose of Flu on the clinical outcome. In this study, to compare the impact of Flu doses in RIC or RTC, we retrospectively analyzed clinical outcomes in patients who received FB or FM conditioning by using nationwide registration data of Japan Society for Hematopoietic Cell Transplantation. Method: Patients aged 16 years of older with acute myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome, chronic myeloid leukemia and malignant lymphoma, who received FB or FM conditioning regimen, and who underwent bone marrow transplantation from an unrelated donor between 2007 and 2016 were included in this study. We excluded patients who received oral Bu. Also, patients who had renal dysfunction in Hematopoietic Cell Transplantation-Comorbidity Index was excluded because Flu dose needs an adjustment due to renal function. As a result, 2809 patients were included in this study. All transplantation data in the present study were obtained from the Transplant Registry Unified Management Program (TRUMP), which included clinical data of hematopoietic cell transplantation performed in Japan. Result: In the FB group (n=1647), high-dose Flu (180 mg/m2; HFB) and low-dose Flu (150/125 mg/m2; LFB) were used in 1334 and 313 patients, respectively. The probabilities of 3-year OS and PFS in the HFB group were significantly higher than that in the LFB group (49% vs 38%, P<0.001; 44% vs 35%, P=0.007). The cumulative incidences of relapse at 3 years were 30.4% and 36.6% (P=0.058), and NRM at 3 years were 25.1% and 28.1% (P=0.24) in the HFB and LFB groups, respectively. In multivariate analysis for OS and relapse, Flu doses was identified as an independent prognostic factor (HR 0.82, P=0.03; HR 0.8, P=0.043). In the FM group (n=1162), high-dose Flu (180 mg/m2; HFM) and low-dose Flu (150/125 mg/m2; LFM) were used in 118 and 1044 patients, respectively. There was no significant difference in the OS, relapse and NRM rates between HFM and LFM (at 3 years, 51.4% vs 48.5%, P=0.596; 22.4% vs 27.0%, P=0.362; 30.7% vs 30.3%, P=0.783). Conclusion: In this large study, we suggested that high-dose Flu was associated with favorable outcome in the FB group, but not in the FM group. Prospective studies and further investigations are needed to clarify the benefit of high-dose Flu in the setting of FB regimen. Disclosures Ozawa: Kyowa-Hakko Kirin: Honoraria; Astellas Pharma Inc.: Honoraria; Pfizer Japan Inc.: Honoraria; Novartis: Honoraria. Kanda:Daiichi Sankyo Company: Honoraria; Bristol-Meyers Squib: Honoraria; Celgene: Honoraria; MSD: Honoraria; JCR Pharmaceuticals: Honoraria; Takeda: Honoraria; Otsuka: Honoraria; Kyowa Hakko Kirin: Honoraria; Chugai: Honoraria; Astellas: Honoraria; Novartis: Honoraria; NextGeM Incorporation: Patents & Royalties: 2019-011392. Atsuta:Kyowa Kirin Co., Ltd: Honoraria; CHUGAI PHARMACEUTICAL CO., LTD.: Honoraria.

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