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)

Patients with primary metastatic or recurrent rhabdomyosarcoma (RMS) have a very poor prognosis. Since high-dose chemotherapy (HDC) +/- TBI was thought to improve survival, many centers performed this therapy using different types of hematopoietic rescue (auto BM or PBSC, allo BM). This is a retrospective, multi-center analysis of the results of treatment in 36 patients with primary metastatic or relapsed RMS who were given HDC +/- TBI and hematopoietic rescue between 1986 and 1994. The median age was 6 years (< 1-22 years). Primary therapy was given according to either one of the Cooperative German Soft Tissue Sarcoma Studies CWS-81, -86, -91 or the European Study for Stage IV Malignant Mesenchymal Tumors in Childhood. There were 22 alveolar RMS, 13 embryonal RMS and one undifferentiated sarcoma. The indication for HDC was primary metastatic disease (27 patients) or a relapse of a primary localized tumor (nine patients). Thirty-two patients were in 1st or 2nd CR when given HDC and four in VGPR. The median time from last event to HDC was 44 weeks (21-110). HDC consisted of fractionated melphalan ((4 x 30-45 mg/m2), VP16 40-60 mg/kg, carboplatin 3 x 400-500 mg/m2) in 26 patients, 10 of whom received additional FTBI. Seven patients were treated with melphalan alone or in combination with carboplatin. Two patients received cyclophosphamide/busulphan with TLI (total lymphoid irradiation) and one cyclophosphamide with FTBI. Thirty-one patients were given autologous BM or PBSC as hematopoietic rescue and five allogeneic bone marrow from HLA-identical siblings. Fourteen patients received GM-CSF or G-CSF after hematopoietic stem cell transfusion (HSCT). Ten patients received adjuvant IL-2. There was one toxic HDC-related death. Nine patients are alive and free of disease with a median observation time of 57 months (32-108). The median time from HDC to relapse was 4 months (1-17). The tumor recurred in the majority of patients at previously known sites; in three cases new metastatic sites were observed. Patients with primary localized tumors who had been treated with HDC because of relapse did slightly better (four of nine alive with NED) than patients with primary metastatic disease (five of 27 alive with NED). HDC is still of uncertain value in the therapy of poor-risk rhabdomyosarcoma and should be performed only as part of controlled clinical trials.
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PMID:Do patients with metastatic and recurrent rhabdomyosarcoma benefit from high-dose therapy with hematopoietic rescue? Report of the German/Austrian Pediatric Bone Marrow Transplantation Group. 902 50

Nine children from 10 to 76 months (median 28.0), weighing 8.5 to 19.7 kg (median 13.0 kg) underwent peripheral blood stem cell separation (PBSCS) or peripheral blood mononuclear cell separation (PBMNCS), after insertion of a double-lumen central venous catheter (8-10 French). Separations were performed with a continuous flow blood separator (Fen-wall CS 3000 plus), running a specially adopted separation-program. In 7 children (5 with neuroblastoma IV, 1 with multifocal Ewing's sarcoma, and 1 with rhabdomyosarcoma IV), stem cells were mobilized by application of G-CSF at a dosage of 15-27.7 micrograms/kg body weight (median 16.25) subcutaneously following high-dose chemotherapy, according to the disease-related protocols, whereas 2 children had PBMNCS to induce graft vs. leukemia (GvL)-reaction in the HLA-identical sibling suffering from relapsed chronic myelogenous leukemia (CML: n = 1), or chronic myelomonocytic leukemia (CMML: n = 1) after allogeneic BMT. In all cases, the collecting procedure was performed after filling the cell separator with priming solution consisting of 2 U of irradiated and washed packed red cells, 250 ml human albumin, and 0.9% NaCl. In the 7 patients with solid tumors between 0.45 and 62.7 x 10(6) CD-34 positive cells/kg body weight were separated; the patient who had the lowest yield was separated twice after another mobilizing course. Three patients (2 with neuroblastoma IV and 1 with multifocal Ewing's-sarcoma) underwent a double transplantation with 1-3 portions of the collected stem cells within a 5- to 6-week interval. Two children had a rapid engraftment on both peripheral blood stem cell transplantations (PBSCTs). The third child, who had the lowest yield and was separated twice had prompt engraftment at the first PBSCT but delayed and incomplete engraftment at the second PBSCT. One patient after adoptive immunotransfer with PBMNCs for relapsed CML is now 40 months in complete cytogenetic and molecular biological remission, whereas the other patient treated for relapsed CMML did not respond to the PBMNC-transfusion. The results indicate that PBSCS and PBMNCS can be performed in children with a body weight below 20 kg.
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PMID:Feasibility of peripheral blood stem cell (PBSC) and peripheral blood mononuclear cell (PBMNC) separation in children with a body weight below 20 KG. 918 Sep 13

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

The hematopoietic colony-stimulating factors have been introduced into clinical practice as additional supportive measures that can reduce, but not eliminate infectious complications associated with therapy-induced neutropenia. Over the past decade, we have begun to appreciate the subtler aspects of the proper use of G-CSF and GM-CSF, identifying appropriate indications and contraindications. In the course of evaluating the multitude of studies, a set of formal recommendations have been propagated for the judicious use of these expensive growth factors. To prevent serious infection, the use of G- or GM-CSF is recommended in a subset of pediatric cancer patients shortly after receiving chemotherapy or a marrow transplant. Children with intensive chemotherapy (e.g., children with high risk ALL, NHL or metastatic neuroblastoma) seem to benefit from hematopoietic growth factors whereas it is not clear that this applies to children undergoing therapy for solid tumors such as rhabdomyosarcoma or Ewing's sarcoma. An exciting development is the use of G-CSF and GM-CSF to mobilize peripheral-blood progenitor cells. Future studies in pediatric cancer patients are clearly warranted to address several issues. Prospective clinical trials are still needed to define specific treatment groups who can benefit from growth factor support. In this regard, efforts must be directed at better defining the endpoints and in particular, assigning value to reduction in treatment of possible infectious complications, such as days in hospital, antibiotic usage and costs. In addition, randomized studies are required to evaluate the proper dosage and duration of therapy, which most likely will vary between groups, depending upon underlying malignancy and therapy given. In addition, combinations of different growth factors have to be tested, particularly if ex vivo expansion and the storage of hematopoietic stem cells are to be utilized in a wider spectrum of patients.
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PMID:[Hematopoietic growth factors in the prevention of infections complications in children with hematologic-oncologic diseases]. 1157 68

There have only been a few reports and limited performance of peripheral blood stem cell (PBSC) collection in very small children weighing less than 10 kg. In this study, we intended to evaluate the safety and yield of PBSC collection, with the efficacy of PBSC transplantation (PBSCT) in the smallest children with solid tumors. From January 1998 to February 2004, 173 children underwent PBSC collection in Samsung Medical Center, Korea. Of these, 15 (8.7%) children weighed less than 10 kg and their clinical diagnoses were neuroblastoma (10 cases), rhabdoid tumor (2 cases), rhabdomyosarcoma (2 cases), and Wilms tumor (1 case). PBSCs were collected following chemotherapy plus G-CSF mobilization. The median age and weight at the time of apheresis were 15 months and 9 kg, respectively. The median number of PBSC collection procedures per case was 4 (range, 2-7). The median cell yield per apheresis product was 0.95 (range, 0.01-33.32) x 10(6)/kg CD34+ cells and 1.96 (range, 0.12-23.39) x 10(8)/kg mononuclear cells. No complications associated with citrate toxicity and other adverse effect were observed during the procedures. After high-dose chemotherapy, 14 patients were reinfused with PBSCs alone and all showed successful hematopoietic recovery. We concluded that PBSC collection would be a safe and practical procedure, even when done in the smallest children, provided that adequate intravascular fluid volume and circulating red cell mass were maintained. Also, the use of PBSCs to support high-dose chemotherapy was well tolerated and might enhance hematological recovery in the smallest children showing the excellent efficacy of PBSCT.
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PMID:Autologous peripheral blood stem cell collections in children weighing less than 10 Kg with solid tumors: experience of a single center. 1589 91