2008-11-01
Topotecan distribution in an anephric infant with therapy resistant bilateral Wilms tumor with a novel germline WT1 gene mutation
Publication
Publication
Cancer Chemotherapy and Pharmacology , Volume 62 - Issue 6 p. 1039- 1044
The therapeutic strategy for bilateral Wilms tumor (WT) remains a challenge. Especially in cases with chemotherapy resistant disease, bilateral nephrectomy is sometimes inevitable. For optimal cure rates stage V WT patients benefit from adjuvant treatment; however, there are limited data available on chemotherapy pharmacokinetics in anephric children. In this report, we describe a 10-month old girl with bilateral Wilms tumor and a novel germline WT1 gene mutation. This patient hardly showed any response on preoperative chemotherapy, and ultimately, underwent sequential bilateral tumor-nephrectomy. Subsequently, during peritoneal dialysis, she received topotecan as adjuvant chemotherapy based on plasma levels, indicating that this is a reasonable option as adjuvant treatment in therapy-resistant Wilms tumor patients after bilateral nephrectomy. This case showed a novel germline WT1 gene mutation of which the correlation with resistant phenotype has to be confirmed in larger cohorts of WT patients.
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doi.org/10.1007/s00280-008-0694-x, hdl.handle.net/1765/29750 | |
Cancer Chemotherapy and Pharmacology | |
Organisation | Erasmus MC: University Medical Center Rotterdam |
Lugtenberg, R., Cransberg, K., Loos, W., Wagner, A., Alders, M., & van den Heuvel-Eibrink, M. (2008). Topotecan distribution in an anephric infant with therapy resistant bilateral Wilms tumor with a novel germline WT1 gene mutation. Cancer Chemotherapy and Pharmacology, 62(6), 1039–1044. doi:10.1007/s00280-008-0694-x |