Steineck Angela, MacKenzie John D, Twist Clare J
Department of Pediatrics, Stanford University School of Medicine, Stanford, California.
Department of Radiology, University of California at San Francisco, San Francisco, California.
Pediatr Blood Cancer. 2016 Nov;63(11):2050-3. doi: 10.1002/pbc.26124. Epub 2016 Jul 11.
Retinoid therapy has contributed to improved outcomes in neuroblastoma. Clinical trials of fenretinide report favorable toxicity and disease stabilization in patients with high risk (HR) neuroblastoma. Skeletal effects have been described with other retinoids, but not with fenretinide to date. Two patients with HR, metastatic, refractory neuroblastoma received protracted courses of oral fenretinide for more than 5 years' duration. Both developed premature long bone physeal closure, causing limb length discrepancies; their neuroblastoma remains in remission. The radiographic and clinical findings reported suggest these skeletal abnormalities may be a consequence of treatment with 13-cis-retinoic acid (13cisRA) followed by prolonged oral fenretinide exposure.
维甲酸疗法有助于改善神经母细胞瘤的治疗效果。芬维A胺的临床试验报告称，高危（HR）神经母细胞瘤患者的毒性反应良好且病情稳定。其他维甲酸类药物曾有骨骼方面的影响描述，但迄今为止芬维A胺尚未出现此类情况。两名患有HR转移性难治性神经母细胞瘤的患者接受了为期5年多的长期口服芬维A胺治疗。两人均出现长骨骨骺过早闭合，导致肢体长度差异；他们的神经母细胞瘤仍处于缓解状态。报告的影像学和临床发现表明，这些骨骼异常可能是先接受13-顺式维甲酸（13cisRA）治疗，随后长期口服芬维A胺的结果。
