Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0035412 (rhabdomyosarcoma)
 6,156 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

A tumor in the left thigh of a 1-year-old boy was diagnosed as rhabdomyosarcoma (stage 1). The tumor was excised and vincristine (1.5 mg/m2 given twice), actinomycin D (15 micrograms/kg given five times), and cyclophosphamide (600 mg/m2 given once) were prescribed. On the 12th day after initiating this chemotherapy, his liver rapidly enlarged and ascites, pleural effusion, and disturbance of consciousness due to hepatic failure successively appeared. Before these clinical signs appeared, laboratory data demonstrated enhanced coagulation and fibrinolysis such as thrombocytopenia refractory to platelet transfusion and a marked increase in fibrinogen and fibrin degradation product (FDP). Hepatic veno-occlusive disease (VOD) was suspected, and based on the laboratory data indicating enhanced coagulation, anticoagulant (gabexate mesylate; FOY), diuretic, and supportive therapy for hepatic failure was begun. The clinical course was favorable and recovery was complete in 2 months, with no sequelae. We propose that platelet count, coagulation factor levels, and FDP may be useful for the early detection of VOD, and an anticoagulant drug such as FOY is worthy of consideration for the prevention and treatment of VOD.
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PMID:Veno-occlusive disease of the liver after combined adjuvant chemotherapy for a 1-year-old boy with rhabdomyosarcoma: potential usefulness of the gabexate mesylate (FOY). 161 37

We describe the case of a 3-year-old girl who developed veno-occlusive disease of the liver while receiving chemotherapy for parameningeal rhabdomyosarcoma. After suffering lethargy and oral mucosal bleeding for one day, the patient exhibited a sudden weight gain and refractoriness to platelet transfusions. Symptoms rapidly worsened with elevation of liver enzymes, bleeding diatheses, and respiratory failure. An ultrasound scan of the liver demonstrated reversed flow in the portal vein. Maximal supportive care, including tracheal intubation and mechanical ventilation, was required. The patient gradually recovered with no respiratory and minimal neurological sequelae. Veno-occlusive disease of the liver should be considered in children receiving chemotherapy who develop weight gain, a sudden drop in platelet count and derangement of liver enzymes. Aggressive supportive measures should be instituted if necessary, as patients surviving the acute phase can expect to make a full recovery.
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PMID:Veno-occlusive disease of the liver after chemotherapy for rhabdomyosarcoma: case report with a review of the literature. 756 14

We report on a 17-year-old young man with rhabdomyosarcoma in the right parotid area. Relapse therapy was performed with high dose chemotherapy and consecutive autologous stem cell rescue. During this therapy heparin-induced thrombocytopenia was diagnosed. Because of its proven antithrombotic activity Danaparoid-Sodium, a natural low molecular glycosaminoglycan preparation, was used for further antithrombotic prophylaxis. We discharged our patient from the laminar air flow unit six months ago. The alternative antithrombotic therapy was tolerated without any problems. No bleeding events occurred, thrombotic complications and veno-occlusive disease of the liver were avoided.
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PMID:[Heparin-induced type II thrombocytopenia within the scope of high dose chemotherapy with subsequent stem cell rescue]. 962 42

We conducted a prospective pilot study to assess the feasibility and safety of high-dose busulfan/melphalan as conditioning therapy prior to autologous PBPC transplantation in pediatric patients with high-risk solid tumors. From January 1995 to January 1999, 30 patients aged 2-21 years (median 8) were entered into the study. There were 14 females and 16 males. Diagnoses included neuroblastoma in 10 patients; Ewing's sarcoma and peripheral neuroectodermal tumor (PNET) in 15 patients and rhabdomyosarcoma in five patients. Treatment consisted of busulfan 16 mg/kg, orally over 4 days (from days -5 to -2) in 6 hourly divided doses, and melphalan at a dose of 140 mg/m2 given by intravenous infusion over 5 min on day -1. G-CSF mobilized PBPC were used as autologous stem-cell rescue. One patient developed a single generalized convulsion during busulfan therapy. The most relevant non-hematologic toxicity was gastrointestinal, manifesting as grade 2-3 mucositis and diarrhea in 12 patients. Two patients died of procedure-related complications, one from veno-occlusive disease of liver and multiorgan failure and the other from adult respiratory distress syndrome. Probability of treatment-related mortality was 6.6 +/- 4.5%. With a median follow-up of 18 months (range, 1-48), 19 patients are alive and disease-free, the actuarial EFS at 4 years being 55 +/- 12% for the whole group. We conclude that high-dose busulfan/melphalan for autologous transplantation in children with solid tumors is feasible even in small patients. It is well-tolerated, with an acceptable transplant-related mortality and has proven antitumor activity.
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PMID:High-dose busulfan/melphalan as conditioning for autologous PBPC transplantation in pediatric patients with solid tumors. 1064 2

Actinomycin-D (Act-D) is a rare cause of veno-occlusive disease (VOD). Between 1993 and 1998, we managed 6 patients, all male, median age 19 months (range 6-48 months) who received Act-D for Wilms' tumour (n=4), clear cell sarcoma (n=1) or rhabdomyosarcoma (n=1). VOD presented with a median platelet count of 12 x 10(9)/l, INR 3.8, fibrinogen 16 mg/l, fibrinogen degradation products (FDPs) > or =80 microg/l, aspartate aminotransferase (AST) 6922 IU/l, bilirubin 47 micromol/l. In 3 cases, transient liver dysfunction and thrombocytopenia without neutropenia had been observed after a previous course of Act-D. All six children developed encephalopathy, hepatomegaly, ascites, reversed portal flow and renal impairment. All received mechanical ventilation and two required haemofiltration. The treatment was supportive. Severe Adult Respiratory Distress Syndrome developed in 3 patients, all of whom died. 3 patients recovered. The outcome of VOD with multi-organ failure is poor. Intravascular coagulopathy precedes and characterises severe VOD during Act-D treatment.
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PMID:Veno-occlusive disease with multi-organ involvement following actinomycin-D. 1137 45

Actinomycin D is an anti-cancer drug commonly used in the treatment of paediatric malignancies such as Wilms' tumour, Ewing's sarcoma and rhabdomyosarcoma. Despite its long history of clinical use, little is known about the pharmacokinetics of actinomycin D in humans, largely due to problems in developing an analytical assay with the required sensitivity to measure relevant clinical concentrations. As actinomycin D treatment in children with cancer is associated with veno-occlusive disease (VOD), and as the dose intensity of actinomycin D treatment has been defined as a significant risk factor for the development of this potentially life-threatening hepatic toxicity, pharmacokinetic studies of actinomycin D may be beneficial in optimizing treatment with this drug. In order to investigate this issue, we developed a sensitive liquid chromatography-mass spectrometry (LC-MS) method for the determination of actinomycin D in human plasma samples. Extraction of analytical samples was carried out with acetonitrile and analysis performed on an API 2000 LC/MS/MS using an internal standard of 7-aminoactinomycin D. A limit of quantitation of 1.0 ng/ml was determined, allowing the reliable measurement of actinomycin D in plasma samples obtained from patients receiving this drug clinically. The method demonstrated good reproducibility, over the calibration curve range of 1.0-100 ng/ml, with intra- and inter-assay precision CVs of 2.7-11.3 and 2.3-7.8%, respectively. Accuracy data showed relative errors of 2.0-16.4 and 10.4-15.2% for intra-assay (n=10) and inter-assay (n=7) experiments, respectively. Initial results of actinomycin D pharmacokinetics in paediatric patients are shown.
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PMID:Determination of anti-cancer drug actinomycin D in human plasma by liquid chromatography-mass spectrometry. 1452 28

Although veno-occlusive disease of the liver is a well-known complication of high-dose chemotherapy and bone marrow transplantation, it has rarely been observed in children who receive conventional chemotherapy. Most cases in the literature consists of children with Wilms tumor. It has been very uncommon in rhabdomyosarcoma patients until recently, although they commonly receive similar anticancer agents. Here the authors report a 2-year-old boy with rhabdomyosarcoma who developed veno-occlusive disease while receiving VAC (vincristine, actinomycin D, cyclophosphamide) chemotherapy regimen according to the IRS-IV protocol. The patient gradually recovered during 2 weeks with supportive treatment only.
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PMID:Veno-occlusive disease in a child with rhabdomyosarcoma after conventional chemotherapy: report of a case and review of the literature. 1809 52

Hepatic veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome, is rapidly progressing and involves life-threatening complications that can occur in patients receiving chemotherapy and/or bone marrow transplantation. No completely satisfactory treatmentstrategies have yet been established. We present a case of rhabdomyosarcoma ina 21-month-old boy who developed pancytopenia, dyspnea, jaundice, massive ascites and body weight gain of more than 10% after receiving conventional chemotherapy. Hepatic VOD was diagnosed. He recovered after supportive care and treatment with high-dose methylprednisolone.
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PMID:Treatment with high-dose methylprednisolone for hepatic veno-occlusive disease in a child with rhabdomyosarcoma. 1905 20

Hepatic Sinusoidal Obstruction Syndrome (HSOS), the new name given to veno-occlusive disease (VOD) of the liver, is a well-known complication of high-dose chemotherapy employed with hematopoietic stem cell transplantation, but it has rarely been observed in children who receive conventional chemotherapy. HSOS following standard chemotherapy has been reported in patients receiving vincristine, actinomycin D, and cyclophosphamide for the treatment of Wilms tumor and more rarely rhabdomyosarcoma. We report a 14-year-old boy with high risk medulloblastoma treated with craniospinal radiation followed by chemotherapy, who experienced severe HSOS after only one course of chemotherapy including carboplatin, vincristine, and cyclophosphamide. To our knowledge, this is the second report of HSOS after standard dose chemotherapy for brain tumor in childhood.
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PMID:Hepatic Sinusoidal Obstruction Syndrome in a child after chemotherapy for medulloblastoma. 1970 18

Hepatic sinusoidal obstruction syndrome (HSOS), also known as veno-occlusive disease, is a well-recognized toxic complication after autologous and allogeneic hematopoietic stem cell transplant, during treatment of Wilms tumor and rhabdomyosarcoma associated with actinomycin-D, and during acute lymphoblastic leukemia therapy due to oral 6-thioguanine. However, its occurrence in the context of chemotherapy regimens for other childhood malignancies is rare. We report a 5-year-old girl with high-risk anaplastic medulloblastoma, who developed severe HSOS during her second cycle of maintenance chemotherapy, consisting of vincristine, cisplatin, and cyclophosphamide. She was treated with defibrotide with complete resolution of the HSOS. These findings and a review of the literature, highlight the occurrence of HSOS in children outside the established settings of hematopoietic stem cell transplantation, Wilms tumor, rhabdomyosarcoma, and acute lymphoblastic leukemia.
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PMID:Hepatic sinusoidal obstruction syndrome during chemotherapy for childhood medulloblastoma: report of a case and review of the literature. 2427 42


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