ABSTRACT
<p><b>OBJECTIVE</b>Hematopoietic stem cell transplantation is currently a unique curative therapy for beta-thalassemia major. However, only 30% of patients have HLA-identical siblings to serve as donors. This study investigated the feasibility of hematopoietic stem cell transplantation from HLA mismatched related donors for beta-thalassemia major in children.</p><p><b>METHODS</b>Between November 2001 and November 2007, 10 patients with beta-thalassemia major at median ages of 4.4 years (range:1.6-9.4 years) received 11 transplantations from their haploidentical donors, either HLA mismatched sibling umbilical cord bloods (n=6) or parents marrows (n=4) or sibling marrow (n=1). The conditioning regiment included fludarabine (100 mg/m2), busulfan (16 mg/kg), cyclophosphamide (200 mg/kg) and antithymocyte globulin.</p><p><b>RESULTS</b>Of the 10 patients, 6 (60%) had sustained engraftment and red blood cell transfusion independence; 2 patients showed transient engraftment but rejected the graft quickly; 1 patients had no evidence of engraftment and developed aplastic anemia; 1 patient who received two transplantations had no evidence of engraftment and developed persistent aplastic anemia. All eight engrafted patients showed grade I to III acute graft-versus-host disease (GVHD), and only one developed limited skin chronic GVHD. The probability of overall and disease-free survival was 90% and 60%, respectively, with a median follow-up duration of 57.1 months (range: 2.5 to 85.1 months).</p><p><b>CONCLUSIONS</b>Haploidentical stem cell transplantation is an alternative option for children with beta-thalassemia major, particularly when a matched sibling donor is not available.</p>
Subject(s)
Child , Child, Preschool , Female , Humans , Infant , Male , Follow-Up Studies , Graft vs Host Disease , Haplotypes , Hematopoietic Stem Cell Transplantation , Histocompatibility Testing , beta-Thalassemia , TherapeuticsABSTRACT
Objective To research the possible influence of operation and human chorionic gonadotropin(HCG) therapy on the development of antisperm antibody (AsAb) and serum levels of estradiol (E_2) and testosterone (T) of cryptorchidism boys after orchiopexy. Methods Forty cryptorchidism boys were studied who were divided into HCG treatment group and without HCG treatment group. Twenty boys who underwent inguinal operation were studied as the operation control group and 20 normal boys as the control group. The serum were collected for detection AsAb, E_2,and T. Results There were no significant difference in serum levels of E_2 and T among the three groups(P 0.05 ). Conclusions The level of AsAb is associated with the destroy of blood barrier and the disorder of immune modulation and endocrine. Treatment with HCG has no effect on AsAb level.
ABSTRACT
<p><b>OBJECTIVE</b>The beta-thalassemia major is a common hereditary hematology disease in southern China. The combination of blood transfusion and iron chelation is now the reference treatment. The allogeneic hematopoietic stem cell transplantation is the only curative therapy for beta-thalassemia major. In this study the investigators observed and evaluated the effects of umbilical cord blood transplantation (UCBT) for patients with beta-thalassemia major.</p><p><b>METHODS</b>Twelve cases of beta-thalassemia major aged from 1.3 to 8.3 years (8 male and 4 female) received UCBT. Eleven of the twelve donors were siblings and one was unrelative. Eight patients received no antigen and four patients received two antigen disparate grafts. According to the Pesaro's classification for thalassemia, 10 patients were at grade I or II, and 2 were at grade III. The HLA-identical patients accepted the conditioning regimen consisting of busulfan, cyclophosphamide and antithymocyteglobulin. The HLA-mismatched patients accepted the conditioning regimen consisting of hypertransfusions, continuous iv desferrioxamine, hydroxyurea, fludarabine, busulfan, cyclophosphamide and antithymocyteglobulin. The harvest stem cells contained 3.63 - 16.0 x 10(7)/kg of nucleated cells, 0.11 - 1.03 x 10(6)/kg of CD(34)(+) cells and 0.17 - 1.18 x 10(5)/kg of colony-forming-unit-granulocyte macrophages. Cyclosporine alone or in combination with mycophenolate mofetil (MMF) was given for acute graft-versus-host disease (aGVHD) prophylaxis.</p><p><b>RESULTS</b>Of the 12 patients, 10 were engrafted. Ten patients had neutrophil recovery (> 0.5 x 10(9)/L) and seven patients had platelet recovery (> 50 x 10(9)/L). The median time was 18.1 and 57.3 days, respectively. Seven patients had disease-free survival (DFS) at a median follow up of 23 months (range 4 - 63 months). Three patients had rejection and autologous hematopoitic reconstitution. Two patients were not engrafted. One patient acquired severe aplastic anemia, another patient died of severe infection. The incidences of grade I and grade II aGVHD were 60% (6/10) and 40% (4/10), respectively. There were no long-term complications in the disease free survivors.</p><p><b>CONCLUSIONS</b>Grade I-II beta-thalassemia major patients receiving sibling UCBT had high DFS. UCBT is an effective way to treat beta-thalassemia major.</p>