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Query: UMLS:C0039730 (thalassemia)
 10,305 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Thalassemia patients can be categorized as class 1 (minimal liver damage and iron overload), class 3 (extensive liver damage from iron overload), and class 2 (intermediate). These categories are prognostic for treatment outcome after marrow transplantation. Class 3 patients have more transplant-related mortality than other patients. This study examines transplantation outcome for class 3 patients. Records were reviewed of 215 patients in class 3 who received transplants in Pesaro from HLA-identical related donors between May 1, 1984 and May 1, 1994. The influence of pretransplant, peritransplant, and posttransplant variables on survival, relapse, and transplant-related mortality was examined by product-limit and proportional-hazards multivariate analysis. Age and conditioning regimen were influential on survival, and regimens with less than 200 mg/kg cyclosporine (CY) were associated with 5-year survival probabilities of .74 and .63 patients younger than 17 years and older patients, respectively. Transfusion history and regimen were influential on rejection with 5 year probabilities of .53 and .24 in patients who received less than or greater than 100 red blood cell transfusions before transplantation and regimens containing less than 200 mg/kg CY. Results of transplantation for patients with advanced thalassemia treatment have improved with the introduction of conditioning regimens with less CY. This has been associated with an increase in rejection (particularly in patients who have received < 100 red blood cell transfusions before transplant). Efforts at reducing the rejection rate by modifying the conditioning regimen should be concentrated on younger patients who have received a small number of transfusions. Patients with thalassemia who have HLA-identical family members should be transplanted before they are in class 3.
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PMID:Marrow transplantation for patients with thalassemia: results in class 3 patients. 863 61

Hereditary disorders represent a very heterogenous group of diseases affecting the hematopoietic system. Bone marrow transplantation is the treatment of choice for immunodeficiencies, mucopolysaccharidosis, thalassemia, Fancoli's anemia, osteopetrosis, and other rare disorders. Indications for bone marrow transplantation are well known when an HLA-identical sibling is identified. Results of alternative donor transplantations are still under investigation. Recently, progress has been made in the analysis of the mechanism of the disease at the molecular level and in the identification of gene mutations. Clinical implications of these discoveries might modify the future indications for bone marrow transplantation.
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PMID:Bone marrow transplantation in children with hereditary disorders. 868 May 13

The European Group of Blood and Marrow Transplantation (EBMT) is collecting information on incidence of bone marrow transplantation in Europe. 203 teams in 26 European countries performed in 1992 a total of 6065 bone marrow transplants (BMT). There were 2666 (44%) allogeneic transplants from 2171 HLA identical sibling donors, 170 non-identical family donors, 29 twin donors and 296 unrelated volunteer donors. There were 3399 autologous transplants (56%): 2494 autologous BMT, 644 autologous peripheral blood stem cell transplants and 261 combined autologous bone marrow peripheral blood stem cell transplants. Transplant indications were leukemias in 2963 patients (49%) (1987 allogeneic, 976 autologous), lymphoproliferative disorders in 1890 patients (31%) (201 allogeneic, 1689 autologous), solid tumors in 739 patients (12%) (10 allogeneic, 739 autologous), aplastic anemia in 194 patients (3%) (193 allogeneic, 1 autologous), thalassemia in 128 patients (2%) (all allogeneic), inborn errors in 115 patients (2%) (all allogeneic) and miscellaneous disorders in 36 patients (32 allogeneic, 4 autologous). Main differences were observed in the number of transplanting teams, and in the number of allogeneic and autologous transplants performed in the different European countries compared to the number of inhabitants.
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PMID:Incidence of bone marrow transplantation in Europe. Report from the European Group for Blood and Marrow Transplantation. 874 19

Thalassemia is widely distributed throughout the world and is one of the major public health problems. The use of bone marrow transplantation, the only curative therapy for thalassemia, is limited because less than 30% of the patients have unaffected and HLA-identical siblings as donors. Cord blood stem cells, an alternative source of stem cells for transplantation, have been successfully transplanted into patients with several diseases after myeloablative therapy. Twenty cord blood samples from unaffected neonates whose siblings had severe thalassemia were collected. The median volume was 80 ml. The median number of cells and colony forming units-granulocyte-macrophage in cord blood was 9.2 x 10(8) and 3.4 x 10(5), respectively. Four of 20 cord blood samples had HLA-matched to the affected siblings. One patient underwent cord blood transplantation with success; one patient is waiting for transplantation.
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PMID:Collection of cord blood stem cells for transplantation in thalassemic patients. 874 91

Bone marrow transplantation was performed on 14 Chinese patients with transfusion dependent thalassaemia major (n = 13) and haemoglobin H disease (n = 1). The donors were HLA identical siblings. The source of haematopoietic stem cells were from bone marrow (n = 13) and umbilical cord blood (n = 1). The pre-transplant conditioning regimens were (1) busulphan 14 mg/kg and cyclophosphamide 200 mg/kg in two patients; (2) busulphan 16 mg/kg, cyclophosphamide 200 mg/kg and anti-thymocyte globulin 110 mg/kg in five patients; (3) busulphan 16 mg/kg, cyclophosphamide 150 mg/kg and anti-thymocyte globulin 110 mg/kg in seven patients. Graft-versus-host disease prophylaxis was cyclosporin A and methotrexate. All patients engrafted and achieved stable haematopoiesis except the one who underwent the umbilical cord blood transplant, who had autologous marrow recovery. One patient who had stable engraftment rejected the marrow graft and developed aplastic anaemia 4 months after BMT. This patient had a second BMT but rejection recurred again. She eventually died of septicaemia. The other 12 patients were transfusion independent and disease free. The majority have gone back to school or work. Disease-free and actuarial survival probability were 85 and 93%, respectively with a median follow-up time of 30 months (13 to 42 months). Our data suggest that BMT from HLA identical siblings for transfusion dependent thalassaemia gives a high chance of cure with acceptable mortality and morbidity, and that a more immunosuppressive pre-transplant conditioning schedule may be required to prevent rejection.
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PMID:A more immunosuppressive pre-transplant conditioning may be required for Chinese patients with thalassaemia. 880 92

Bone marrow transplantation from an HLA-identical sibling can cure thalassaemia. The risk of chemotherapy-induced sterility, however, represents a deterrent for many patients already at risk of gonadal insufficiency and reduced fertility because of the effects of transfusional iron overload. We report here the first patient transplanted for thalassaemia, after ablative therapy with busulfan and cyclophosphamide, who, despite late pubertal maturation, became pregnant and delivered a full-term, normal infant.
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PMID:Successful pregnancy after bone marrow transplantation for thalassaemia. 883 25

Fetus-to-fetus transplantation has been suggested for the treatment of hemoglobinopathies in utero. However, dissimilar results have to date been obtained by different groups. We describe a case in which fetus-to-fetus transplantation in HLA-identical twins was performed at the 19th week of gestation by infusion of 0.8 ml of fetal blood from normal to beta-thalassemia affected fetus with the main aim of inducing tolerance. No evidence of engraftment, determined by KM19 polymorphism, was present after 2 years of the procedure. Moreover, an alloreactive cytotoxic T lymphocyte precursor (CTLp) study of affected fetus vs donor and other different stimulators showed that immunization vs tolerance was the real effect of the procedure.
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PMID:Evidence of induced non-tolerance in HLA-identical twins with hemoglobinopathy after in utero fetal transplantation. 887 30

Langerhans' cell histiocytosis (LCH) is an uncommon disorder of childhood, formerly referred to histiocytosis X. A significant proportion of children with disseminated disease may undergo progression to a fatal outcome despite chemotherapy with single or multiple agents. Only six cases of LCH treated with BMT have been reported in the literature, including two cases of autologous BMT. Of them, only one was less than 14 years of age. We describe a 4-year-old child whose disseminated, refractory Langerhans' cell histiocytosis was not controlled by front-line monotherapy with etoposide, nor by rescue treatment with combined chemotherapy (vinblastine and etoposide) and immunotherapy (steroids and cyclosporine). Due to the high risk of fatal progressive disease, he underwent bone marrow transplantation from his HLA-identical sister who was heterozigous for beta-thalassemia. On day 24 after transplantation marrow reconstitution was evident, with WBC count 2.3 x 10(9)/L, neutrophil count > 0.5 x 10(9)/L, and platelet count 72 x 10(9)/L. Engraftment was demonstrated by PCR DNA analysis. The patient was discharged on day 25. After transplantation he experienced fever for 11 days and developed signs of grade I cutaneous and intestinal graft-versus-host disease, that was treated with methylprednisolone from days 11 to day 68 (1 mg/kg/day for 18 days, then tapered). He became transfusion independent on day 24; the hemoglobin value was 7.5 g/dL on day 54 and has remained > 10 g/dL since day 200. Features of heterozygous beta-thalassemia have been evident since then. Bone marrow aspirate was normal on days 25 and 94. At the time of this writing he remains in excellent condition, disease and treatment free, 25 months after transplantation. Although limited, current experience suggests that bone marrow transplantation has the potential to cure refractory Langerhans' cell histiocytosis.
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PMID:Bone marrow transplantation for refractory Langerhans' cell histiocytosis. 895 63

We analyzed risk factors in 724 patients evaluable for acute graft-versus-host disease (GVHD) and in 614 patients evaluable for chronic GVHD who had received bone marrow transplantation (BMT) from HLA-identical siblings and/or parents for thalassemia and/or microdrepanocytosis, in a single institution. The overall incidence of grade II-IV and III-IV acute GVHD (aGVHD) was 26.9% and 13.5%, respectively. The cumulative incidence of grade II-IV aGVHD in patients treated with cyclosporine (CsA)/methylprednisolone (MP) or CsA/methotrexate (MTX)/MP was 32% and 17%, respectively (P=0.001). In logistic regression analysis, the risk factors associated with the onset of grade II-IV aGVHD in the entire group of patients were: patient age < or = 4 years (P=0.009), male patient sex (P=0.023), GVHD prophylaxis with CsA/MP or MTX/MP (P=0.000), more than twofold elevated alanine aminotransferase (P=0.001), and patient seropositivity for two to three herpes viruses (P=0.007). In patients treated with CsA/MP, splenomegaly > 2 cm (P=0.042) and donor age > or = 17 years (P=0.034) predicted aGVHD. Risk factors for grade III-IV aGVHD were similar to the risk factors identified for grade II-IV aGVHD. Moreover, moderate and severe liver fibrosis or cirrhosis predicted grade III-IV aGVHD (P=0.018). The incidence of chronic GVHD (cGVHD) was 27.3%. The probability of cGVHD at 2 years after BMT in patients with grade 0, I, II, and III-IV aGVHD was 15%, 32%, 53%, and 54%, respectively. Among patients with absent or grade I-IV aGVHD, prior aGVHD (P=0.000), female donor sex (P=0.000), use of alloimmune female donors for male patients (0.009), and GVHD prophylaxis with CsA/MP or MTX/MP (P=0.003) predicted cGVHD. This data should be considered in clinical management and in future investigations for improvement of immunosuppressive prophylaxis in BMT patients with thalassemia.
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PMID:Graft-versus-host disease after bone marrow transplantation for thalassemia: an analysis of incidence and risk factors. 908 26

A 12-year-old girl with beta-thalassemia hemoglobin E disease received a marrow transplant from her HLA-identical elder brother in July 1995. She had previously been treated by repeated blood transfusions. Conditioning included busulfan 16 mg/kg for 2 days and cyclophosphamide 120 mg/kg for 2 days. Cyclosporine was used for graft-versus-host disease prophylaxis. Spiking fevers occurred on days 6 and 11. Plasmodium falciparum parasites, both trophozoites and gametocytes, were found on the peripheral blood smear. Quinine 30 mg/kg three times a day for 7 days followed by a single dose of mefloquine 25 mg/kg was given. The fever subsided within 2 days and parasitemia cleared in 4 days. After transplant, the girl autologously reconstituted and was followed-up over 15 months.
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PMID:Malaria infection after allogeneic bone marrow transplantation in a child with thalassemia. 920 23


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