Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: EC:2.1.1.69 (BMT)
 2,655 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Retrospectively we analyzed the histocompatibility data and clinical results of bone marrow transplantation in 51 patients who received marrow from unrelated donors (UD) from 1977 to 1987 at one of four UK BMT centers. We compared the results with those obtained in 51 transplants carried out at the same centers using HLA-identical (ID) sibling donors. Of the UD/recipient pairs 32 (63%) were serologically identical for HLA A, B, and DR antigens, and 37% showed varying degrees of mismatch. UD-BMT primary diagnoses were: severe aplastic anemia or Fanconi's anemia (n = 17), acute leukemia (n = 11), chronic myeloid leukemia (n = 21), and other conditions (n = 2). T cell depletion of the graft was associated with a significant improvement in survival in both UD and ID-BMT. Graft failure was more common in recipients of UD than of ID transplants (13 [25%] vs. 5 [10%] P = 0.05) but there was no significant difference in the frequency of acute or chronic graft-versus-host disease. Actuarial survival was superior for recipients of ID transplants (UD vs. ID: 49% vs. 78%, respectively, at 3 months; 32% vs. 63% at one year). Reduced survival for recipients of UD-BMT was confirmed in case control regression analysis (relative risk 3.0, P = 0.01). Nevertheless in patients whose only alternative is a partially mismatched family donor we think that UD-BMT is justified.
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PMID:Unrelated donor marrow transplantation between 1977 and 1987 at four centers in the United Kingdom. 218 Jan 50

We report a case of a 19-year-old male with congenital aplastic anemia and multiple abnormalities; short stature, hypoplastic thumb, skin pigmentation and mental retardation. He was admitted to our hospital because of severe pancytopenia. Bone marrow aspiration showed markedly hypocellular marrow with 42% myeloblasts. He was diagnosed as AML (M2) transformed from Fanconi's anemia and underwent allo-BMT from an HLA-identical father. The conditioning regimen consisted of high dose Ara-C, high dose etoposide and 12Gy fractionated total body irradiation. Severe toxicity associated with the conditioning regimen was not observed. Cyclosporin A and short-term methotrexate were administered for prophylaxis of acute GVHD. Neither acute nor chronic GVHD were observed. He is well and free of disease for 15 months since BMT. Very few cases of Fanconi's anemia with leukemic transformation treated by BMT have been reported. Long-term observation will be necessary to evaluate our conditioning regimen for Fanconi's anemia with leukemic transformation.
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PMID:[Allogenic bone marrow transplantation for Fanconi's anemia with leukemic transformation from an HLA identical father]. 764 54

Although renal wasting of phosphate is relatively common, Fanconi's syndrome following ifosfamide chemotherapy is rare. This case illustrates the possibility of developing Fanconi's syndrome despite the apparent lack of toxicity during previous ifosfamide exposure. As the use of high-dose ifosfamide-containing regimens prior to BMT increases, the occurrence of this adverse effect may become more frequent. Morbidity due to Fanconi's syndrome can be decreased by close monitoring and aggressive management of fluid and electrolytes.
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PMID:Fanconi's syndrome due to ifosfamide. 843 14

Allogeneic bone marrow transplantation represents the treatment of choice for severe bone marrow failure in patients with Fanconi's anaemia (FA). In view of the increased sensitivity to alkylating agents documented in this condition, much attention has focused on reducing the conditioning chemotherapy. We present a 13-year-old girl in whom sibling allogeneic BMT after conditioning with low-dose cyclophosphamide only resulted in graft rejection. However, a second transplant using the same donor proved successful following a more intensive conditioning regimen. This case demonstrates the phenotypic variability of FA, and highlights the need for tailoring the conditioning regimen for a given patient.
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PMID:Successful second bone marrow transplant for Fanconi's anaemia following escalation of conditioning. 933 38

Although patients with Fanconi's anaemia (FA) exhibit a heightened sensitivity to DNA cross-linking agents, modified doses of CY continue to be used in their conditioning prior to BMT. We measured the pharmacokinetics and metabolism of CY in six children with FA using an established high performance thin layer chromatography technique. CY doses ranged between 5 and 20 mg/kg (median 10 mg/kg). The median CY clearance was 0.6 l/h/m2 (range 0.4-1.1 l/h/m2), t1/2 was 8.1 h (range 6.7-9.5 h) and volume of distribution was 0.19 l/kg (range 0.16-0.34 l/kg), respectively. These results contrast with those previously reported from a comparable group of non-FA children in whom the median CY clearance was 3.2 l/h/m2 (range 2-5 l/h/m2) (P = 0.035), t1/2 was 2.4 h (range 2-3.8 h) (P = 0.035) and volume of distribution 0.5 l/kg (range 0.26-0.95 l/kg) (NS). Unlike the control group in whom the presence of inactive metabolites of CY was common, metabolites could not be found in any FA patient. The enhanced sensitivity of children with FA to CY may in part result from altered drug metabolism.
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PMID:Cyclophosphamide metabolism in children with Fanconi's anaemia. 1045 39

We report a unique case of brucellosis transmitted by BMT. An 8-year-old boy with the diagnosis of Fanconi's anemia received an allogeneic BMT from his HLA-identical sibling. Routine culture from the infused marrow suspension grew Brucella abortus on day +4 post BMT. Spiking fevers occurred on days +2 and +16. The first febrile episode responded to broad-spectrum antibiotic therapy. However, the second episode did not. B. abortus was isolated from blood cultures taken during the second febrile episode. The Brucella agglutination titer was negative. Antibiotic therapy with oral doxycycline and i.v. gentamycin was successful with no recurrence of infection during 13 months of follow-up. The donor's blood culture was also positive for B. abortus and Brucella antibodies were detectable at 1:320 titer when he presented with fever and hepatosplenomegaly on day +32. We emphasize the need to consider brucellosis in patients undergoing BMT. We suggest that donor and recipient be evaluated for brucellosis especially in countries where the incidence of this infection is relatively high.
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PMID:Brucellosis transmitted by bone marrow transplantation. 1091 36

Post-transplant lymphoproliferative disorder (PTLD) is uncommonly of T cell origin, especially following BMT. We describe a 13-year-old boy with severe aplastic anemia (SAA) and no evidence of Fanconi's anemia who underwent BMT at 11 years of age using CY 10 mg/kg once daily i.v. on days -5, -4, antilymphocyte globulin (ALG) 30 mg/kg once daily i.v. on days -5 approximately -3 and CsA from day -1 as conditioning. The BMT failed and he received a further peripheral blood stem cell transplant (PBSCT) 240 days after BMT. Conditioning was with CY 50 mg/kg once daily i.v. on days -5 approximately -2, and ALG 15 mg/kg once daily i.v. on days -4 approximately -2. GVHD prophylaxis included CsA and MTX. Engraftment was later confirmed by cytogenetic studies. Desquamation and ulcers of the oral mucosa and mouth angle developed in the 13th month post PBSCT. A buccal mucosa biopsy on day +524 revealed only plasmacytosis. Immunosuppressants were discontinued at that point. Generalized lymphadenopathy, prolonged fever (waxing and waning) and facial swelling developed in the 18th month post PBSCT. A neck lymph node biopsy on day +601 showed T cell lymphoma of diffuse large cell type with monoclonal TCR gamma-chain gene rearrangement. A FISH study showed that the malignant T cells were of recipient origin. EBV in situ hybridization was negative. He did not receive further treatment apart from discontinuation of immunosuppressants. He was followed up in our out-patient clinic and showed good performance 1170 days post PBSCT. We speculate that a different mechanism was operating in the pathogenesis of T cell lymphoma in this case. Risk factors include SAA and two transplants, conditioned with CY and ALG, long term use of CsA and treatment with azathioprine.
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PMID:T cell lymphoproliferative disorder following bone marrow transplantation for severe aplastic anemia. 1108 91

Fanconi's anemia (FA) is an autosomal-recessive disorder characterized by a progressive pancytopenia, diverse congenital abnormalities and increased predisposition to malignancy. Sixteen children with FA, aged between 4 to 16 were divided into two groups according to treatments. Nine children had bone marrow transplantation and seven children were treated with steroid and/or anapolan. The changes in dental caries, caries-associated microflora, salivary status and periodontal health were investigated in children with FA. Data were analyzed by one-way ANOVA. A statistically significant difference was found in hematological findings between children who have received bone marrow transplantation (BMT+) and the others, who have not received (BMT-). There was no significant difference in dental caries experience, salivary flow rate, buffering capacity, mutans streptococci and Lactobacilli levels between the study groups. A statistically significant difference was found in gingival index, plaque index, bleeding on probing, probing depth scores between the patients with FA in BMT(+) and BMT(-) groups (p<.05). In conclusion, besides systemic control, additional preventive measures during their whole life to maintain oral health is necessary in these children.
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PMID:The effect of bone marrow transplantation on systemic and oral health in Fanconi's aplastic anemia. 1149 16

Androgens widely used in the treatment of bone marrow failure syndromes can in rare cases cause hepatic peliosis, a pathological entity characterized by multiple blood-filled cavities in the liver parenchyma. Bone marrow failure syndromes per se are associated with a low coagulation status, which is further magnified by bone marrow transplantation for aplastic anaemia due to deep thrombocytopenia. Both these conditions can cause bleeding; their combination is especially dangerous. We describe two cases of aplastic anaemia due to paroxysmal nocturnal hemoglobinuria and Fanconi syndrome, in which patients developed peliosis hepatis after prolonged treatment with androgens. One patient developed severe subcapsular bleeding, successfully treated with catheterization of the right hepatic artery and embolization of the bleeding site. The second patient bridged over deep post-transplant aplasia with high frequency platelet transfusions, and demonstrated an uncomplicated post-BMT course. We suggest avoiding or interrupting treatment with androgens in patients preparing for BMT.
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PMID:Peliosis hepatis following treatment with androgen-steroids in patients with bone marrow failure syndromes. 1802 86