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Response of metastatic recurrent neuroblastoma to nitisinone: A modulator of tyrosine metabolism
Author(s) -
Kobrinsky Nathan L.,
Sjolander Diane E.
Publication year - 2006
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.20420
Subject(s) - medicine , neuroblastoma , endocrinology , metabolite , cyclophosphamide , chemotherapy , cancer research , oncology , biology , genetics , cell culture
Nitisinone blocks the tyrosine pathway and may be effective in treating neuroblastoma. A 33‐month‐old male with heavily treated metastatic, recurrent, N‐MYC amplified neuroblastoma received nitsinone (0.8 mg/kg/day escalated to 5.0 mg/kg/day). Dramatic tumor regression and resolution of pain without toxicity were observed. At 10 weeks, the tumor progressed. Nitisinone, low dose cyclophosphamide and doxorubicin subsequently produced a very good partial response. At 18 months the disease progressed. The child succumbed 21 months after starting nitisinone. Nitisinone produced an increase in tyrosine and catecholamine metabolite (HVA, VMA, and metanephrines) levels. Nitisinone may be a promising agent in metastatic neuroblastoma. Pediatr Blood Cancer 2006, 46:517–520. © 2005 Wiley‐Liss, Inc.