scholarly journals Long-term results of allogeneic peripheral blood hematopoietic stem cell transplantation for severe aplastic anemia

2020 ◽  
Vol 42 ◽  
pp. 27
Author(s):  
E. Aladag ◽  
H. Goker ◽  
H. Demiroglu ◽  
S. Aksu ◽  
N. Sayınalp ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Peripheral Blood ◽  
Long Term Results ◽  
Hematopoietic Stem

Alternative Donor Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4348-4348
Author(s):  
Meerim Park ◽  
Kyung Nam Koh ◽  
Keun Wook Bae ◽  
Mee Jeong Lee ◽  
Ho Joon Im ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Peripheral Blood ◽  
Hematopoietic Stem ◽  
Matched Sibling

Abstract Abstract 4348 Background Hematopoietic stem cell transplantation (HSCT) from matched sibling donor is the standard first-line treatment for children with severe aplastic anemia (SAA). However, the management of SAA lacking a suitable donor remains a great challenge. For those children, HSCT using unrelated donor or mismatched related donor could be a therapeutic alternative. The purpose of this study is to evaluate the outcome in children with SAA who received HSCT from donors other than matched sibling. Patients and Method Between March 2003 and July 2009, 17 patients received HSCT from alternative donors (AD) at Asan Medical Center. We reviewed their medical records and analyzed their transplant-related parameters and outcome. Results Of a total of 17 patients, 11 were male and the median age at HSCT was 9.0 years, ranging from 3.0 to 16.7 years. Four patients had Fanconi anemia and 13 had acquired SAA including 2 who developed SAA after liver transplantation. Donors included unrelated bone marrow (U-BM) in 5, unrelated peripheral blood (U-PB) in 6, unrelated cord blood (U-CB) in 2 and related haploidentical peripheral blood (H-PB) in 4. Of 17 patients, 15 (88%) achieved sustained engraftment. Of 15 with engraftment, only 1 patient who received HSCT from U-CB died of severe GI GVHD and the other 14 patients remain on stable normal counts without transfusion support. All 2 patients (1 U-BM, 1 H-PB) who failed to engraft were dead despite DLI or 2nd HSCT. With a median follow-up of 31.9 months, the Kaplan-Meier estimated overall survival at 2 years was 76.6%. Conclusion In children with SAA, HSCT from AD including haploidentical family donor could be considered as a treatment option if the patients have no matched sibling donor. Given the limitation of this study such as small number of patients and short follow-up period, further trial will be necessary. Disclosures: No relevant conflicts of interest to declare.

Haploidentical Hematopoietic Stem Cell Transplantation for Acquired Severe Aplastic Anemia

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3227-3227 ◽  
Author(s):  
Miao Miao ◽  
Xiang Zhang ◽  
Ting Xu ◽  
Song Jin ◽  
Hong Wang ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Peripheral Blood ◽  
Hematopoietic Stem ◽  
Acquired Severe Aplastic Anemia

Abstract Objective: To evaluate the efficacy and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in patients with acquired severe aplastic anemia (SAA), who lacked suitable related or unrelated HLA-matched donors. Methods: 39 SAA patients underwent haplo-HSCT from Jul 2012 to Jun 2015 at our center. There were 23 males and 16 females at a median follow-up of 11 (range, from 0 to 36) months. The median time from diagnosis to transplantation was 1 (range, from 0.5 to 52) months. The median ages of SAA patients and related haploidentical donor were 23 years (range, 9 to 51years) and 45 (range, from 21 to 61) years, respectively. All patients were given BuCy plus ATG conditioning regimen. GVHD prophylaxis regimen consisted of cyclosporine A (CsA), mycophenolate motetil (MMF), and short-term methotrexate. Results: Stem cells were collected from bone marrow in 23.08% (n=9) of patients, peripheral blood in 2.56% (n=1), bone marrow plus peripheral blood in 74.36% (n=29) patients. 36 patients received haplo-HSCT combined with the third part of cord blood transfusion 92.31%. The median stem cell dose transplanted was 9.76 (range, from 4.02 to 20.10)×108/kg for mononuclear cells, while 3.4 (range, from 1.05 to 8.60)×108/kg for CD34 cells. 36 patients achieved neutrophil engraftment at a median of 12 (range, from 9 to 28) , and 29 patients achieved platelet engraftment at a median of 29 (range, from 10 to 26) days. Cumulative incidence of III°~IV° acute graft versus host disease (aGVHD) was 8.9±4.9%. 6 patients died of transplant-related mortality (TRM), including 4 from severe infection, and 1 from TMA. The overal survival rate of all patients was 83.2%±6.4% Conclusions: Haplo-HSCT is likely to be an option for SAA patients without suitable related or unrelated HLA-matched donors, in consideration of the acceptable TRM and severe GVHD incidences. Disclosures No relevant conflicts of interest to declare.

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