The Desmoid Tumor Research Foundation

September 2018

Evidence of hydroxyurea activity in children with pretreated desmoid-type fibromatosis: A new option in the armamentarium of systemic therapies.

Author Information

Ferrari A(1), Orbach D(2), Affinita MC(3), Chiaravalli S(1), Corradini N(4), Meazza C(1), Bisogno G(3), Casanova M(1).

(1)Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milano, Italy. (2)SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, Paris, France. (3)Pediatric Hematology and Oncology Division, Padova University, Padova, Italy. (4)Institut d'Hematologie et d'Oncologie Pediatrique, Centre Leon Berard, Lyon, France.

Publication

Ir Med J

Summary

The treatment paradigm in desmoid-type fibromatosis (DF) has changed in recent years from a surgery-based strategy to a multidisciplinary approach that includes systemic therapies. Among various medical therapies, hydroxyurea has been considered of potential interest. This case series summarizes the experience gained at four centers using hydroxyurea in relapsing DF. Hydroxyurea was given orally at an initial dose of 20 mg/kg/day (escalated up to 30 mg/kg/day as necessary, if well tolerated). The response rate was 18.7% major partial remissions, 37.5% considering any amount of shrinkage, 68.7% considering symptom response or signs of tissue response as well. In patients with no progression, the treatment was continued for 9-24 months. The response rate was moderate, but similar to that reported for other medical therapies currently considered as treatment options in this disease. Though further, larger series are needed to confirm as much, hydroxyurea has potential as an effective alternative therapy for DF.

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