Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0039730 (thalassemia)
 10,305 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Cyclosporin A (CsA) has been shown to be useful in the prophylaxis of acute graft-versus-host-disease (GVHD). However, this immunosuppressive agent produces multiple side-effects including nephrotoxicity, hypertension, hypertricosis, gum hyperplasia, infections, and neurotoxicity. We report a retrospective analysis of neurotoxicity in 625 recipients transplanted for thalassemia and given CsA as part of GVHD prophylaxis. Neurotoxicity consisted in mental status changes, tremor, headache (grade 1), visual disturbance and cortical blindness (grade 2) and seizures and coma (grade 3). The overall toxicity was 28.8% and the incidence of convulsions was 10.1%. Neurological findings were reversible after temporary reduction or discontinuation of CsA. Class 3 patients, when prepared with protocol 6 (Bu 14 + Cy 200 and CsA for GVHD) or when they developed acute GVHD, had the highest risk of convulsions. Age, sex, different conditioning regimens, different anticonvulsive prophylaxis, liver damage due to iron-overload and/or to chronic inflammation did not influence the occurrence of CsA-related CNS toxicity. The occurrence of acute GVHD with concomitant use of high-dose corticosteroids is the single significant predisposing factor in the occurrence of convulsions. Grades 1 and 2 of neurotoxicity occurred earlier and were not influenced even by acute GVHD.
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PMID:CsA-associated neurotoxicity and ineffective prophylaxis with clonazepam in patients transplanted for thalassemia major: analysis of risk factors. 883 9

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

Cyclosporin A is an essential drug for graft versus host disease (GvHD) prophylaxis in allogeneic hematopoietic stem cell transplantation. Cyclosporin A-associated neurotoxicity is common but poorly understood. The authors herein report on 3 patients receiving transplants for thalassemia. GvHD prophylaxis included cyclosporin A, prednisolone, and methotrexate. All patients developed a status epilepticus with cortical signal alterations located mainly in occipital regions on T2-weighted magnetic resonance imaging. Complete disappearance of the neurologic symptoms and radiographic findings was observed without discontinuing cyclosporin A therapy. Especially, pediatric thalassemics receiving busulfan/cyclophosphamide as preparative regimen seem to be significantly prone to cyclosporin A-associated seizures.
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PMID:Cyclosporin A-associated status epilepticus related to hematopoietic stem cell transplantation for thalassemia. 1463 24

Allogeneic SCT remains the only potential cure for patients with thalassemia. However, most BMT candidates lack a suitable family donor and require an unrelated donor (UD). We evaluated whether BMT using UDs in high-risk adult thalassemia patients can offer a probability of cure comparable to that reported employing an HLA-compatible sibling as donor. A total of 27 adult thalassemia patients (15 males and 12 females, median age 22 years) underwent BMT from a UD selected by high-resolution HLA molecular typing. The conditioning regimen consisted of Busulphan (BU, 14 mg/kg) plus Cyclophosphamide (CY, 120 or 160 mg/kg) in 12 cases and BU (14 mg/kg), Thiotepa (10 mg/kg) and CY (120-160 mg/kg) in the remaining 15 cases. Cyclosporine-A and short-term Methotrexate were used for graft-versus-host disease (GVHD) prophylaxis. In all, 19 patients (70%) are alive and transfusion-independent after a median follow-up of 43 months (range 16-137). A total of 10 patients (37%) developed grade II-IV acute GVHD and six (27%) chronic GVHD. Eight patients (30%) died from transplant-related causes. UD-BMT can cure more than two-thirds of adult thalassemia patients, and is a particularly attractive option for patients who are not compliant with conventional treatment.
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PMID:Unrelated donor stem cell transplantation in adult patients with thalassemia. 1620 30