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)

Between 1985 and 1989, eight children underwent two successive bone marrow transplantations. The initial disease was chronic myelomonocytic leukemia in three patients, chronic myelocytic leukemia in two, acute M7 nonlymphoblastic leukemia in one, sickle cell anemia in one, and thalassemia major in one. The preparation in view of the second grafting included high-dose chemotherapy in all patients, associated with antithymocytic globulin transfusion and total nodal irradiation in three patients. Hematological recovery was similar after both graftings. Infectious complications were not more common following the second graft than after the first one. On the other hand, the rates of rejection and graft-versus-host disease were lower, probably due to a more intensive immunosuppressive therapy. The prognosis of chronic leukemia relapsing after a first graft does not seem to be improved by a second attempt.
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PMID:Second bone marrow transplantation in eight children. 146 68

Consideration of a bone marrow transplant (BMT) for a child with a genetic disease depends upon many factors including the pathophysiology of the disorder, the natural history of the disease, whether an alternative therapy exists and whether a donor is available. Children with disorders such as severe combined immunodeficiency disease (SCID), in which life expectancy is minimal, are obviously candidates for a BMT, even with less than optimal donors, while those with disorders such as beta-thalassemia major, in which an alternative therapy exists, must be considered more carefully and only with an optimal donor. The risks of conditioning therapy, graft-versus-host disease (GVHD), and early death as well as the cost are critical to this decision and must be viewed in light of the potential outcome of a successful BMT and the life expectancy and quality of life with a BMT. For some genetic diseases with multisystem involvement (e.g., Hurler's mucopolysaccharidosis), the efficacy of a BMT has been reasonably demonstrated, providing significant brain damage has not occurred previously. For some other storage-related diseases, there is no place for BMT. Further studies are essential to increase our knowledge as to its potential role in other types of genetic-associated diseases.
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PMID:Bone marrow transplantation for the treatment of genetic diseases. 195 29

Three patients with acute myeloblastic leukemia, thalassemia major and aplastic anemia experienced hemorrhagic cystitis on the 23rd, 35th and 36th day respectively after allogeneic bone marrow transplantation. The conditioning regimens before transplantation comprised cyclophosphamide with or without busulfan and total lymphoid irradiation. The hematuria lasted from 5 to 45 days and then subsided after treatment. Multiple factors including the toxicity of chemotherapeutic agents, viral infection and graft-versus-host reactions may contribute to late onset hemorrhagic cystitis after allogeneic bone marrow transplantation. Adequate treatment to minimize urothelial damage from chemotherapy, screening for viral infection and controlling graft-versus-host disease are mandatory in decreasing the complication of late-onset hemorrhagic cystitis after bone marrow transplantation.
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PMID:Late onset hemorrhagic cystitis after allogeneic bone marrow transplantation. 255 95

The incidence of interstitial pneumonitis (IP) was reviewed in 80 consecutive patients who received allogeneic T lymphocyte-depleted bone marrow transplantation (BMT) for malignant and non-malignant diseases. Pretransplant conditioning used in malignant disorders included total lymphoid irradiation (TLI) 150 cGy x 4, total body irradiation (TBI) 200 cGy x 6, and cyclophosphamide (CY) 120 mg/kg. In non-malignant diseases conditioning included no TBI, but adjusted doses of TLI in addition to CY (severe aplastic anemia) or CY and busulfan (severe beta-thalassemia major). In the malignant group only one patient developed graft-versus-host disease (GVHD) grade I; IP developed in 12 out of 61 patients (19.7%) and IP-associated fatality occurred in five patients (8.2%). Cytomegalovirus (CMV) was associated with only two of the five fatal IP. In the non-malignant group there was no GVHD; one patient out of 19 (5.2%) had IP, which was fatal and not associated with CMV. These data indicate that fatal IP may appear in the absence of GVHD. The relatively low incidence of IP-related mortality in recipients of allogeneic T lymphocyte-depleted BMT suggests that although prevention of GVHD and elimination of drugs used for GVHD prevention may reduce the incidence of fatal pulmonary complications, other approaches have to be investigated for complete prevention of IP which still represents a major complication in patients with malignant hematologic disorders treated by allogeneic BMT.
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PMID:Interstitial pneumonitis in T cell-depleted bone marrow transplantation. 285 Aug 31

The present report summarizes our experience in applying a new approach in bone marrow transplantation for the treatment of beta-thalassemia major. Ex-vivo pretransplant T-lymphocyte depletion with CAMPATH-1 was used for prevention of acute and chronic graft versus host disease and total lymphoid irradiation was added for the conditioning regimen for abrogation of potential rejection of T-cell depleted marrow allografts. Ten patients with homozygous beta-thalassemia major were 9-48 months of age (median 18.5 months) and received HLA-identical allogeneic T-cell depleted marrow after treatment with total lymphoid irradiation, busulfan and cyclophosphamide. Seven patients are alive and free of disease, 3-46 months post-transplantation. The actuarial probability of survival and of disease-free survival at two years was 70%. Three patients died: one of intracranial hemorrhage post-transplantation, one from busulfan interstitial pneumonitis, and one who rejected the first graft and developed fatal chronic graft versus host disease after a second transplant. Seven patients are alive and well with follow-up of 3-45 months, with no signs of acute or chronic GVHD. We conclude that T-cell depleted bone marrow transplantation is indicated for homozygous transfusion dependent young patients with beta-thalassemia who are minimally transfused, particularly in areas where optimal conventional therapy is not feasible.
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PMID:Bone marrow transplantation in beta-thalassemia major. The Israeli experience. 306 78

The Authors describe a case of severe obstructive-restrictive pulmonary disease following bone marrow transplantation in a Cooley disease affected child. It is an uncommon complication, strictly related to chronic Graft Versus Host Disease (c-GVHD) and for the prevention of which it is may be necessary a more aggressive c-GVHD immunosuppressive therapy. Bone marrow transplantation represents today the only actual resolutive therapy for thalassemia major; so the number of transplantations and related undesirable effects are thought to be increasing in the future. Therefore, a permanent Centre for Control of the complications can be useful for the improvement of survival and quality of life in these patients.
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PMID:[Obstructive-restrictive pulmonary syndrome as long-term side effect of bone marrow transplantation in thalassemia major. Prognostic and therapeutic problems]. 761 75

Even though there is recognized morbidity and death associated with bone marrow transplantation, this procedure has been performed successfully in a substantial number of patients with hemoglobinopathies. However, finding a suitable related donor is often difficult and the morbidity associated with the use of unrelated donors is high. Several reports indicate that fewer than 30% of patients with thalassemia major and fewer than 20% of patients with sickle cell anemia have histocompatible siblings. Human umbilical cord blood (UCB) contains hematopoietic stem cells capable of reconstituting bone marrow. To date, approximately 200 transplantations have been performed with UCBs. Early results suggest that, even with substantial human leukocyte antigen (HLA) incompatibility, a decrease in the incidence of graft-versus-host disease occurs with cord blood. The extent to which HLA incompatibility can be tolerated when cord blood is used has not been determined. These results raise the possibility that UCB obtained from unrelated donors could be used for transplantation in patients with hemoglobinopathies. This review summarizes current data on UCB stem cells used for transplantation in hematologic diseases. The review contains a discussion of the potential uses of UCB for patients with hemoglobinopathies and the value of programs designed to collect UCB from newborn infants with hemoglobinopathies, from siblings of patients with hemoglobinopathies, and from groups of ethnic minorities similar to those in which hemoglobinopathies are found.
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PMID:Umbilical cord blood stem cells: application for the treatment of patients with hemoglobinopathies. 915 73

Graft-versus-host disease (GVHD), graft rejection, disease recurrence and long-term toxicity remain significant obstacles to successful allogeneic bone marrow transplantation (BMT) in children with genetic diseases. In an attempt to improve results, we used a preparative regimen consisting of three alkylating agents, busulfan (BU), thiotepa (TTP) and cyclophosphamide (CY), for T cell-depleted allogeneic bone marrow transplantation instead of the conventional BU-CY protocol. The effect of this intensified regimen was investigated in 26 consecutive children with genetic diseases who underwent T cell-depleted BMT from HLA-identical siblings. Sixteen patients were males and 10 females, of median age 5 (0.2-14) years. The diseases included beta-thalassemia major, osteopetrosis, severe combined immunodeficiency, Wiskott-Aldrich syndrome, familial agranulocytosis, congenital idiopathic hemolytic anemia (CIHA), Gaucher's disease, Niemann-Pick disease, Hurler's syndrome, and adrenoleukodystrophy. The conditioning regimen consisted of BU 4 mg/kg x 4 days (-8 to -5), TTP 5 mg/kg x 2 days (-4 and -3), and CY 60 mg/kg x 2 days (-2 and -1). Engraftment was as expected, with WBC >1.0 x 10(9)/l at day +19 (10-33), ANC >0.5 x 10(9)/l at day +22 (10-56) and platelets >25 x 10(9)/l at day +32 (18-131). Transplant-related mortality was 19%. Overall survival and disease-free survival (DFS) at 60 months follow-up were both 77%. Our results with the BU-TTP-CY regimen followed by T cell-depleted BMT in genetic diseases may provide a basis for prospective comparison with the standard conditioning regimen of BU-CY in the management of children suffering from these conditions.
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PMID:The role of thiotepa in allogeneic bone marrow transplantation for genetic diseases. 1033 39

Between October 1995 and October 1998, 24 children aged 9 months to 17 years (median 11 years) underwent cytokine-mobilized allogeneic peripheral blood stem cell (PBSC) transplantation for treatment of hematological disorders. All of the transplants were the first allogeneic transplant for the recipient. Twenty patients were transplanted for hematological malignancies (ALL = 8, AML = 6, CML = 4, MDS = 2) and four patients were transplanted for non-malignant disease (thalassemia major = 2, Wiskott-Aldrich syndrome = 1, Kostmann's syndrome = 1). Nineteen donors were HLA-identical siblings, four were HLA-matched or single antigen mismatched parents, and one was a syngeneic transplant. Donors aged 8 to 38 years (median 15 years, 14 donors <18 years) received G-CSF 10 microg/kg/day subcutaneously beginning 4 days before PBSC collection and were submitted to one to three leukapheresis collections. The median CD34+ cell yield was 7.8 x 106 cells/kg recipient body weight. All patients achieved an ANC >0.5 x 109/l after a median of 13 days (range 10-21). Twenty-three patients eventually achieved platelet transfusion independence. One patient died on day 63 without ever achieving platelet transfusion independence. Four patients received platelet transfusions to maintain a platelet count well above 20 x 109/l due to bleeding complications. Of the 19 evaluable patients, the median time to a non-transfused platelet count of 20 x 109/l was 12 days (range 0-44). Ten of 23 at-risk patients developed acute GVHD grades II to IV, with grades III to IV in four patients. Twelve of 19 patients followed for at least 100 days have developed chronic GVHD (extensive = 2, limited = 10) with an actuarial risk of chronic GVHD of 75% at 1 year. The Kaplan-Meier estimate of event-free survival is 65% at 2 years. Four patients died (GVHD = 3, VOD = 1), three patients relapsed, and one patient with thalassemia major had a late graft failure with autologous recovery. Based upon our experience, allogeneic PBSCT is safe for both pediatric donors and recipients and engraftment of neutrophils and platelets is rapid. Bone Marrow Transplantation (2000) 25, 13-18.
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PMID:Cytokine-mobilized allogeneic peripheral blood stem cell transplants in children result in rapid engraftment and a high incidence of chronic GVHD. 1065 8

Using a non-myeloablative, immunosuppressive, fludarabine-based conditioning regimen, we performed allogeneic peripheral blood stem cell transplants totally on an outpatient basis in four patients (two with chronic myelogenous leukemia, one with acute myelogenous leukemia and one with thalassemia major). The median granulocyte recovery time to 0.5 x 109/l was 10 days and the lowest absolute neutrophil count was 0.064 x 109/l; only one patient developed thrombocytopenia below 20 x 109/l. No patient required red blood cell transfusions and one was given a single prophylactic platelet transfusion. All patients are alive at 210-390 (median 285) days and have definite evidence of chimerism; one developed biopsy-proven GVHD on day 50, with a limited cutaneous rash. The procedure is less costly than its counterpart using myeloablative conditioning regimens and may represent another approach in the management of patients requiring an allogeneic stem cell transplant. Bone Marrow Transplantation (2000) 25, 131-133.
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PMID:Hematopoietic stem cell allografts using a non-myeloablative conditioning regimen can be safely performed on an outpatient basis: report of four cases. 1067 69


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