Response of metastatic recurrent neuroblastoma to nitisinone: A modulator of tyrosine metabolism

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...

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Bibliographic Details
Published in:Pediatric Blood & Cancer 2006-04, Vol.46 (4), p.517-520
Main Authors: Kobrinsky, Nathan L., Sjolander, Diane E.
Format: Article
Language:English
Subjects:
Antineoplastic Agents - adverse effects
Antineoplastic Agents - therapeutic use
Antineoplastic Combined Chemotherapy Protocols - therapeutic use
Biological and medical sciences
Child, Preschool
Combined Modality Therapy
Cyclohexanones - adverse effects
Cyclohexanones - therapeutic use
Disease Progression
Dose-Response Relationship, Drug
Drug Administration Schedule
Enzyme Inhibitors - adverse effects
Enzyme Inhibitors - therapeutic use
Fatal Outcome
General aspects
Humans
HVA
Male
Medical sciences
metanephrine
Neoplasm Recurrence, Local - drug therapy
neuroblastoma
Neuroblastoma - drug therapy
Neuroblastoma - secondary
nitisinone
Nitrobenzoates - adverse effects
Nitrobenzoates - therapeutic use
Remission Induction
Treatment Outcome
Tumors
tyrosine
Tyrosine - drug effects
Tyrosine - metabolism
VMA
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Summary: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.
ISSN:1545-5009
1545-5017
1096-911X
DOI:10.1002/pbc.20420