Patrizia Dall'Igna1, Laurence Brugieres2, Anne Sanlaville Christin2, Rudolf Maibach3, Michela Casanova4, Rita Alaggio5, Jean de Ville de Goyet6, Jozsef Zsiros7, Bruce Morland8, Piotr Czauderna9, Margaret Childs10, Daniel C Aronson11, Sophie Branchereau12, Penelope Brock13, Giorgio Perilongo14. 1. Pediatric Surgery Division, Department of Women's and Children's Health, University of Padua, Padua, Italy. 2. Department of Oncology for Children and Adolescents, Institute Gustave Roussy, Villejuif, Cedez, France. 3. IBCSG Coordinating Center, Effingerstrasse 40, 3008, Berne, Switzerland. 4. Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy. 5. Department of Pathology, Children's Hospital of Pittsburgh of UPCM, Pittsburgh, Pennsylvania. 6. ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad alta specializzazione, Pediatric Surgery, Palermo, Italy. 7. Department of Pediatric Oncology, Emma Children's Hospital, Academic Medical Centre, Amsterdam, The Netherlands. 8. Department of Oncology, Birmingham Children's Hospital, Birmingham, United Kingdom. 9. Department of Surgery and Urology for Children and Adolescents, Medical University of Gdansk, Gdansk, Poland. 10. University of Nottingham, Nottingham, United Kingdom. 11. Department of Pediatric Surgery, Kinderspital Zürich, University of Zürich, Zürich, Switzerland. 12. Department of Pediatric Surgery, Bicêtre Hospital, Paris Sud University, Le Kremlin Bicêtre, Paris, France. 13. Department of Oncology, Great Ormond Street Hospital, NHS Trust, London, WC1N 3JH, United Kingdom. 14. Department of Women's and Children's Health, University of Padua, Padua, Italy.
Abstract
BACKGROUND: The purpose of this study was to evaluate clinical characteristics, treatment, and survival of children, who were diagnosed with hepatoblastoma (HB) in their first 6 months of age, enrolled in the SIOPEL 2 and 3 protocols. METHODS: Seventy-nine patients, treated between 1994 and 2006, were analyzed after stratification into three age groups: <1 month, between 1 and 3 months, and between 3 and 6 months. All received preoperative chemotherapy. RESULTS: Clinical characteristics were similar in both trials: 4 patients had pulmonary metastases at diagnosis, 4 had α-fetoprotein <100 ng/ml, 68 were operated by partial hepatectomy, and 7 received liver transplant. Chemotherapy courses were delayed in 8.5%, 8.4%, and 11.8% of cycles in the three groups. Doses were calculated according to weight for children <5 and 5-10 kg, and further reduced in 18.1%, 6.8%, and 5.9% of cycles. Acute toxicity was manageable. Long-term hearing loss was the major problem at follow-up occurring in two-thirds of children. Ten patients experienced progression or relapse, and 5 of 10 died. After a median follow-up of 5.6 years, the 5-year overall survival (OS) and event-free survival (EFS) were 91% (95% confidence interval [CI]: 84-96%) and 87% (95% CI: 78-92%), respectively. CONCLUSIONS: The 5-year OS and EFS of children <6 months of age affected by HB seem to be similar to those documented in the elder children. Dose reduction does not seem to jeopardize the long-term outcome and may explain the lower toxicity profile. Ototoxicity though appears as high as in the whole population of SIOPEL 2 and 3. The treatment for these children should be further explored in international studies, particularly focusing on prevention of hearing loss.
BACKGROUND: The purpose of this study was to evaluate clinical characteristics, treatment, and survival of children, who were diagnosed with hepatoblastoma (HB) in their first 6 months of age, enrolled in the SIOPEL 2 and 3 protocols. METHODS: Seventy-nine patients, treated between 1994 and 2006, were analyzed after stratification into three age groups: <1 month, between 1 and 3 months, and between 3 and 6 months. All received preoperative chemotherapy. RESULTS: Clinical characteristics were similar in both trials: 4 patients had pulmonary metastases at diagnosis, 4 had α-fetoprotein <100 ng/ml, 68 were operated by partial hepatectomy, and 7 received liver transplant. Chemotherapy courses were delayed in 8.5%, 8.4%, and 11.8% of cycles in the three groups. Doses were calculated according to weight for children <5 and 5-10 kg, and further reduced in 18.1%, 6.8%, and 5.9% of cycles. Acute toxicity was manageable. Long-term hearing loss was the major problem at follow-up occurring in two-thirds of children. Ten patients experienced progression or relapse, and 5 of 10 died. After a median follow-up of 5.6 years, the 5-year overall survival (OS) and event-free survival (EFS) were 91% (95% confidence interval [CI]: 84-96%) and 87% (95% CI: 78-92%), respectively. CONCLUSIONS: The 5-year OS and EFS of children <6 months of age affected by HB seem to be similar to those documented in the elder children. Dose reduction does not seem to jeopardize the long-term outcome and may explain the lower toxicity profile. Ototoxicity though appears as high as in the whole population of SIOPEL 2 and 3. The treatment for these children should be further explored in international studies, particularly focusing on prevention of hearing loss.
Authors: Roberta Angelico; Chiara Grimaldi; Carlo Gazia; Maria Cristina Saffioti; Tommaso Maria Manzia; Aurora Castellano; Marco Spada Journal: Cancers (Basel) Date: 2019-10-31 Impact factor: 6.639
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