26 december 2010: Bron: Pedriatic Blood and Cancer

Wanneer kinderen met een recidief van hun hersenkanker naast zware hoog gedoseerde chemokuren nieuwe autologe stamcellen krijgen (stamcellen zijn van hun eigen bloed gemaakt) dan heeft ca. 25% kans op een langdurige levensverlenging en ook een kans om hun kanker definitief te overwinnen. Maar ook sterft ca. 25% aan de gevolgen van de zware chemokuren. Dat is natuurlijk een groot dilemma. Aan de andere kant als je niets meer doet weet je zo goed als zeker dat je kind zal overlijden. Hier het abstract van de fase I studie. Het volledige studierapport is te koop bij Wiley online Library

Pediatr Blood Cancer. 2010 Dec 1. [Epub ahead of print]

Phase I study of tandem high-dose chemotherapy with autologous peripheral blood stem cell rescue for children with recurrent brain tumors: A pediatric blood and marrow transplant consortium study.

Gilman AL, Jacobsen C, Bunin N, Levine J, Goldman F, Bendel A, Joyce M, Anderson P, Rozans M, Wall DA, Macdonald TJ, Simon S, Kadota RP.

Levine Children's Hospital, Charlotte, North Carolina.

Abstract

BACKGROUND: High-dose chemotherapy with autologous stem cell rescue (HDC/SCR) has produced responses and prolonged survival for some children with recurrent brain tumors, but is associated with considerable morbidity and mortality. A Phase I trial of two cycles of HDC/SCR for recurrent brain tumors in children was performed to determine the maximum tolerated doses for a novel regimen.

PROCEDURES: Two cycles of HDC/SCR were given. Cycle 1 included thiotepa and carmustine given on days -5, -4, and -3. Four to six weeks later, patients received cycle 2 which included thiotepa and carboplatin given on days -5, -4, and -3. Autologous peripheral blood stem cells (PBSC) were infused on day 0 of each cycle.

RESULTS: Thirty-two patients were treated and 25 patients received both cycles of HDC/SCR. Common toxicities included mucositis, emesis, diarrhea, anorexia, and pancytopenia. Eight of 32 (25%) assessable children died from regimen-related toxicity. Pulmonary failure occurred in seven patients. Seven patients had grade 3-4 neurotoxicity. The 3-year event-free survival (EFS) was 25%.

CONCLUSIONS: We determined the maximum tolerated regimen to be thiotepa 600 mg/m(2) and carmustine 300 mg/m(2) followed by thiotepa 600 mg/m(2) and carboplatin 1,200 mg/m(2). Pulmonary toxicity was considerable. The toxic death rate was similar to other trials of HDC/SCR for children with recurrent brain tumors performed during the same time period. The regimen resulted in prolonged time to progression for a significant number of patients and long-term survival for some patients with recurrent medulloblastoma and rhabdoid tumor. Pediatr Blood Cancer © 2010 Wiley-Liss, Inc.

PMID: 21125619 [PubMed - as supplied by publisher]


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