Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0018133 (graft-versus-host disease)
 18,032 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The kinetics of circulating lymphoid cells were evaluated in three children suffering from beta-thalassemia major after HLA-identical sibling placental blood transplant (PBT) in one patient and placental blood plus bone marrow transplantation (BMT) in two patients. Recovery of the main lymphocyte subsets, as determined by phenotype analysis of circulating PBMCs, was complete within 2 months after transplant. NK (CD56+) cells were the first to appear in peripheral blood, followed by T (CD3+, CD2+, CD7+) and B (CD19+) cells. Of the T lymphocytes, the CD8+ were the first to reconstitute, but recovery of CD4+ cells was also rapid and within 6 months these T cells reached normal values. The expression of CD57 by NK or T cells was slightly delayed. The evaluation of RA and RO isoform expression of the CD45 molecule showed a prevalence of the CD45RA antigen with a ratio of 2-3:1. In the PBT only patient, T cells expressing the CD45RO antigen prevailed in the early post-transplant period. Severe or chronic GVHD was not observed. This experience demonstrates that reconstitution of lymphocyte subsets is successful in genetic hematological diseases after transplantation of HLA-identical placental blood or placental blood plus bone marrow from healthy or heterozygous siblings. Bone Marrow Transplantation (2000) 26, 743-747.
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PMID:Lymphocyte subset reconstitution after HLA-identical placental blood transplantation (PBT) or PBT plus bone marrow transplantation (BMT) in three children with beta-thalassemia major. 1104 55

Using nonmyeloablative, immunosuppressive, fludarabine (FLU)-based conditioning regimens, we have performed allogeneic peripheral blood stem cell transplants in 26 patients (8 with chronic myelogenous leukemia, 6 with acute myelogenous leukemia, 10 with acute lymphoblastic leukemia, 1 with myelodysplasia, and 1 with thalassemia major). Conditioning consisted of FLU/busulphan/cyclophosphamide/cyclosporin-A (CyA)/methotrexate, or FLU/melphalan/CyA/methotrexate. The median granulocyte recovery time to 0.5 x 10(9)/l was 11 days, whereas the median platelet recovery time to 20 x 10(9)/l was 12 days. Twelve patients did not need red blood cell transfusions, and 8 did not need platelet transfusions. In 21 individuals (81%), the procedure could be completed fully on an outpatient basis. Follow-up times range between 30 and 600 days: one patient failed to engraft and recovered endogenous hemopoiesis; six out of 26 patients developed acute graft-versus-host disease (GVHD) whereas 7/22 developed chronic GVHD. Twelve patients (46%) have died, nine of them with a relapsing disease and three with GVHD; median post-transplant survival (SV) was 300 days, whereas the 12-month SV was 42%. The 100-day mortality was 3.8% and the transplant-related mortality was 11.5%. This procedure is substantially less costly than its counterpart, using in-hospital myeloablative conditioning regimens, and it may represent another approach in the management of patients requiring an allogeneic stem cell transplant.
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PMID:Results of an outpatient-based stem cell allotransplant program using nonmyeloablative conditioning regimens. 1127 33

We describe our experience of setting up an allogeneic BMT program at the Christian Medical College Hospital, Vellore over a period of 13 years, from October 1986 to December 1999. Two hundred and twenty-one transplants were performed during this period in 214 patients, with seven patients undergoing second transplants. Indication for BMT were thalassemia major - 106 (48%), CML - 30, AML - 35, ALL - 10, SAA - 22, MDS - six and six for other miscellaneous disorders. The mean age of this patient cohort was 15.6 years (range 2-52). Graft-versus-host disease of grades III and IV was seen in 36 patients (17%) and this was the primary cause of death in 20 patients (9.2%). All patients and donors were CMV IgG positive. Sepsis was the primary cause of death in 16 patients (7.4%), 10 bacterial, four fungal and two viral. One hundred and ten of this series of patients are alive and disease free (50%) with a median follow-up of 24 months (range 2-116). These results are comparable to those achieved for patients with similar disease status in transplant units in the Western world and cost a mean of US$15 000.
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PMID:Allogeneic bone marrow transplantation in the developing world: experience from a center in India. 1147 34

Bone marrow transplantation is the only therapeutic option that can eliminate thalassemic disease. Early results indicated that children in class 3 Lucarelli had a much worse outcome because of high nonrejection mortality and high rejection rate. We therefore tried to investigate a nonmyeloablative stem cell transplantation (NST) approach for such a disease in order to reduce mortality and rejection. We report here the case of successful NST in a 10-year-old girl who had class 3 Lucarelli beta-thalassemia major. The conditioning regimen consisted of busulfan, fludarabine, antilymphocyte globulin and total lymphoid irradiation. Her GVHD prophylaxis included mycophenolate mofetil and cyclosporin. The patient had full donor engraftment without acute and chronic GVHD. She is now alive and well and remains disease-free 1 year after transplant.
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PMID:Full chimerism in nonmyeloablative stem cell transplantation in a beta-thalassemia major patient (class 3 Lucarelli). 1223 28

The authors describe a 5-year-old boy with beta-thalassemia major who received bone marrow transplantation (BMT) from a human leukocyte antigen (HLA)-matched unrelated donor. The conditioning regimen consisted of 16 mg/kg busulfan and 200 mg/kg cyclophosphamide. The transplantation was complicated with grade II graft-versus-host disease, although prophylaxis with cyclosporine and short-term methotrexate was carried out. Cytomegalovirus disease occurred at 2 months after transplantation but was controlled successfully. The child remains disease-free and in good clinical condition 53 months after BMT. The authors suggest that BMT from an HLA-matched unrelated donor could be considered as an alternative treatment in patients with beta-thalassemia major when no HLA-matched donor is available.
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PMID:Successful matched-unrelated bone marrow transplantation in a patient with beta-thalassemia major. 1236 1

Hematopoietic cell transplantation (HCT) has been used for more 30 years for the treatment of selected malignant and nonmalignant diseases. Traditionally, HCT for hematological disorders has relied on myeloablative conditioning before HLA-identical sibling bone marrow transplantation to correct the underlying hematological defect. Most children with hematological diseases who are referred to HCT have features that portend significant morbidity and early mortality. Among SAA patients who have HLA-identical sibling donors, younger patients with profound pancytopenia might be considered early for HCT. For others who lack sibling donors, patients who receive HCT from alternate sources have generally failed one or more courses of intensive immunosuppressive therapy and remain transfusion-dependent, some with hemosiderosis, red cell alloimmunization, and platelet transfusion refractoriness [44,46,48]. Currently, HCT for SCD is generally restricted to those who have experienced a significant sickle-related complication such as stroke, recurrent acute chest syndrome, or recurrent painful episodes [7,13]. In contrast, most reserve HCT in thalassemia for younger, Lucarelli class I, good-risk patients who have HLA-identical sibling donors, and veer away from older, high-risk thalassemics for whom transplantation is a riskier clinical intervention. For groups such as young adults with thalassemia major, HCT might become more widely applicable if its toxicity was reduced. Several approaches undergoing development include reduced-intensity conditioning and attempts to prevent GVHD. New methods to reduce the intensity and toxicity of conditioning as well as to use highly purified stem cells with the reduction in graft versus host disease may allow for the use of matched unrelated donors or haploidentical donors. This would serve to provide potentially more children who could benefit from stem cell transplantation with donors. These advances will hopefully lead to benefits for the majority of children who lack HLA-identical donors.
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PMID:New approaches to hematopoietic cell transplantation for hematological diseases in children. 1243 Jun 22

The purpose of this study was to designed to analyze and explore the diagnosis and treatment on a patient with beta-thalassemia major accompanied with intestinal acute graft-versus-host disease (aGVHD) grade IV after allo-peripheral blood stem cell transplantation (allo-PBSCT). The conditioning regimen was consisted of busulfan (18 mg/kg), cyclophosphamide (200 mg/kg), thiotepa (6 mg/kg) and horse anti-thymocyte globulin (110 mg/kg). The nucleated cells (10.6 x 10(9)/kg) and CD34(+) CD38(-) cells (11.7 x 10(6)/kg) were infused. At +14 days PBSCT, ANC and WBC achieved 0.66 x 10(9)/L and 1.4 x 10(9)/L respectively. The engraftment was successful but the patient depended on transfusion. The range of blood WBC was from 1.4 x 10(9)/L to 2.4 x 10(9)/L. The myelogram showed aplastic anemia. Hematopoiesis was restored continually after the second transfusion of the donor's cells. At +11 days the patient was suffered from cutaneous aGVHD grade II. Diarrhea occurred at +33 day when cutaneous aGVHD had been improved, and exaggerated obviously after the second transfusion at +39 days. The serious diarrhea continued for 2 months and intestinal aGVHD grade IV was finally diagnosed. Our conclusion was that intestinal aGVHD possibly occurred even when cutaneous aGVHD was improved. For a patient with refractory diarrhea after allo-PBSCT, intestinal aGVHD was considered first when the engraftment evidence was definited but peripheral blood cells and bone marrow were abnormal.
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PMID:[Clinical Study on A Patient with Intestinal Acute Graft-Versus-Host Disease Grade IV after Allo-Peripheral Blood Stem Cell Transplantation] 1257 95

We report our experience in successful unrelated-donor bone marrow transplantation in 2 cases of beta-thalassemia major, which are approved to be the first 2 cases in Asia. The 2 children receiving transplantation of the bone marrow from unrelated donors were diagnosed as having beta-thalassemia major, whose gene mutation type was homozygous and double heterozygote. High-resolution HLA typing found a mismatch with 1 sub-locus in both cases, and red blood cell type was mismatched in 1 case. Pretransplant conditioning protocol contained busulfan (BU, 16 mg/kg x b.w.), cyclosphamide (CY, 200 mg/kg x b.w.), anti-thymocyte globulin (ATG, 90 mg/kg x b.w.) and fludarabine (25 mg x d-1 x m-2). To prevent graft-versus-host disease (GVHD) episodes, cyclosporine-A (Cs-A) and methotrexate (MTX) were administered. The 2 cases experienced condition resembling serum sickness during pretransplant conditioning. After bone marrow transplantation, 1 case had grade acute GVHD in the skin, another developed grade acute GVHD in both the skin and intestinal tract, but the condition was brought under control in both cases after proper treatment. The time for peripheral neutrophil granulocyte recovery to above 0.5 x 10(9)/L was 19 and 16 days postoperatively, respectively, and the time of WBC recovery to normal was 54 and 80 days postoperatively. Platelet recovery to over 50 x 10(9)/L occurred on postoperative days 61 and 90, and Hb recovered to above 100 g/L in both case on days 110 and 83 respectively. The time of final blood transfusion was 53 and 62 days postoperatively for the 2 patients. Gene mutation type in the 2 cases was switched to normal of the donors. After retrieval of relative literature, the 2 cases were approved as the first 2 successful unrelated-donor bone marrow transplantation cases in Asia. This will provide a new possibility of donor supply for hematopoietic stem cells transplantation in thalassemia.
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PMID:Unrelated bone marrow transplantation for beta-thalassemia major: report of the first 2 successful cases in Asia. 1265 Dec 55

A group of 21 consecutive patients aged 4-20 (median 13) years was prospectively allografted using a reduced intensity preparative regimen. The group included both malignant (acute lymphoblastic leukemia, acute myelogenous leukemia, and chronic myelogenous leukemia) and nonmalignant (aplastic anemia, Diamond-Blackfan anemia, thalassemia major and adrenoleukodystrophy) conditions. Follow-up times ranged between 16 and 1038 days. Four of 21 patients (9.5%) developed acute graft-versus-host disease, and 2 of them died, whereas limited chronic graft-versus-host disease was observed in 2 of 15 cases. The 100-day mortality was 19%. Median overall survival was above 1038 days, whereas the 34-month survival was 55%. These data show that reduced intensity stem cell transplantation in children permits rapid engraftment from siblings with little toxicity.
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PMID:Reduced-intensity stem cell transplantation in children and adolescents: the Mexican experience. 1265 65

A 5-year-old boy with beta-thalassemia major received an unrelated umbilical cord blood transplantation (URD-UCBT). The URD-UCB was six antigen HLA matched. The infused cell dose was 7.5 x 10(7)/kg nucleated cells. Conditioning included busulfan 20 mg/kg, cyclophosphamide 200 mg/kg, fludarabine 150 mg/kg, thiotepa 6 mg/kg, and antithymocyte globulin 90 mg/kg. The post transplant complications were mild hepatic veno-occlusive disease, acute GVHD grade III, and CMV interstitial pneumonia. The subject has been ex-thalassemic for more than 20 months post transplant. The chronic GVHD was limited and could be controlled by methylprednisolone combined with mycophenolate. This is the first successful report of an unrelated umbilical cord blood transplantation for beta-thalassemia major from China.
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PMID:Unrelated umbilical cord blood transplant for beta-thalassemia major. 1272 86


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