TREATMENT
OF SEVERE ��-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 ��-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 ��-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 Hb��110g/L. Gene analysis showed that the severe
��-thalassemia gene mutation of the recipients were transferred to the donor
types.
Conclusion: Severe
��-thalassemia patients with HLA-match or one antigen mis-matched related
donor are recommended for HSCT. This is an effective treatment for the
disease