TREATMENT OF SEVERE Β-THALASSEMIA BY SIBING HAMOTOPOIETIC STEM CELL TRANSPLANTATION : AN ANALYSIS OF 8 CASES

Zhu Weiguo, Wu Jianchun, Yang Ming, Zhang Zhenhong, Zhuang Xiaoqing, Guan Guixian, He Yuelin, Cheng Shaojie

Department of Pediatrics, Nanfang Hospital, Guangzhou, China

 

Objective: To evaluate the clinical outcome of sibling hamotopoietic stem cell transplantation (HSCT) in severe β-thalassemia patients and the affected factors.

Methods: Data of 8 patients received sibling HSCT of bone marrow or cord blood in our hospital in recent two years were retrospectively analyzed. All of them were diagnosed as severe β-thalassemia gene type by PCRand dot hybridization. Five of them were HLA identical, three has one antigen mis-matched, two have different blood type with the donors. Conditioning regemen were BU 16mg/kg+CTX 200mg/kg+ATG 90mg/kg. CsA and MTX were used to prevent Graft versus Host Disease (GVHD).

Results: Seven patients reconstituted hemotopoiesis, five of them were HLA indentical, among which one patient who had different blood type with the donor had to receive the second transplantation by bone marrow after a failure transplantation by peripheral blood hemotopoietic stem cells (PBHSCs). The transfused HSCs in two cases with HLA DR antigen mis-matched get ingraftment, while another case with HLA A antigen mis-matched was rejected. The mean recovery time of peripheral blood were 18 days for WBC 1.0×109/L, 25 days for WBC to normal level; 61days for PLT 50×109/L; 40 days for Hb to 100g/L; and 31 days for the last blood transfusion time. Transplantation-related complication are acute GVHD (3 cases II), chronic GVHD (1 case), VOD (1 case), interstitial pneumonia caused by CMV, mycotic sepsis. The follow-up duration of  seven successful cases was 2~3 years. all of them need no blood transfusion and can keep Hb110g/L. Gene analysis showed that the severe β-thalassemia gene mutation of the recipients were transferred to the donor types.

Conclusion: Severe β-thalassemia patients with HLA-match or one antigen mis-matched related donor are recommended for HSCT. This is an effective treatment for the disease

 
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