Brachytherapy for Pediatric Patients at Gustave Roussy Cancer Campus: A Model of International Cooperation for Highly Specialized Treatments.

PURPOSE: Childhood cancer is rare, and treatment is frequently associated with long-term morbidity. Disparities in survival and long-term side effects encourage the establishment of networks to increase access to complex organ-conservative strategies, such as brachytherapy. We report our experience of an international cooperation model in childhood cancers. METHODS AND MATERIALS: We examined the outcome of all children referred to our center from national or international networks to be treated according to a multimodal organ-conservative approach, including brachytherapy. RESULTS: We identified 305 patients whose median age at diagnosis was 2.2 years (range, 1.4 months to 17.2 years). Among these patients, 99 (32.4%) were treated between 2015 and 2020; 172 (56.4%) were referred from national centers; and 133 (43.6%) were international patients from 31 countries (mainly Europe). Also, 263 patients were referred for primary treatment and 42 patients were referred for salvage treatment. Genitourinary tumors were the most frequent sites, with 56.4% bladder/prostate rhabdomyosarcoma and 28.5% gynecologic tumors. In addition to brachytherapy, local treatment consisted of partial tumor resection in 207 patients (67.9%), and 39 patients (13%) had additional external radiation therapy. Median follow-up was 58 months (range, 1 month to 48 years), 93 months for national patients, and 37 months for international patients (P < .0001). Five-year local control, disease-free survival, and overall survival rates were 90.8% (95% confidence interval [CI], 87.3%-94.4%), 84.4% (95% CI, 80.1%-89.0%), and 93.3% (95% CI, 90.1%-96.5%), respectively. Patients referred for salvage treatment had poorer disease-free survival (P < .01). Implementation of image guided pulse-dose-rate brachytherapy was associated with better local control among patients with rhabdomyosarcoma referred for primary treatment (hazard ratio, 9.72; 95% CI, 1.24-71.0). At last follow-up, 16.7% patients had long-term severe treatment-related complications, and 2 patients (0.7%) had developed second malignancy. CONCLUSIONS: This retrospective series shows the feasibility of a multinational referral network for brachytherapy allowing high patient numbers in rare pediatric cancers. High local control probability and acceptable late severe complication probability could be achieved despite very challenging situations. This cooperation model could serve as a basis for generating international reference networks for high-tech radiation such as brachytherapy to increase treatment care opportunities and cure probability.

Acute Myeloid Leukemia in Adolescents and Young Adults Treated in Pediatric and Adult Departments in the Nordic Countries.

BACKGROUND: Studies on adolescents and young adults with acute lymphoblastic leukemia suggest better results when using pediatric protocols for adult patients, while corresponding data for acute myeloid leukemia (AML) are limited. PROCEDURE: We investigated disease characteristics and outcome for de novo AML patients 10-30 years old treated in pediatric or adult departments. We included 166 patients 10-18 years of age with AML treated according to the pediatric NOPHO-protocols (1993-2009) compared with 253 patients aged 15-30 years treated in hematology departments (1996-2009) in the Nordic countries. RESULTS: The incidence of AML was 4.9/million/year for the age group 10-14 years, 6.5 for 15-18 years, and 6.9 for 19-30 years. Acute promyelocytic leukemia (APL) was more frequent in adults and in females of all ages. Pediatric patients with APL had similar overall survival as pediatric patients without APL. Overall survival at 5 years was 60% (52-68%) for pediatric patients compared to 65% (58-70%) for adult patients. Cytogenetics and presenting white blood cell count were the only independent prognostic factors for overall survival. Age was not an independent prognostic factor. CONCLUSIONS: No difference was found in outcome for AML patients age 10-30 years treated according to pediatric as compared to adult protocols.

Subcutaneous anti-D treatment of idiopathic thrombocytopenic purpura in children.

We investigated the effect of subcutaneous anti-D IgG as platelet enhancing therapy in children with idiopathic thrombocytopenic purpura (ITP). Twenty-three children were treated with subcutaneous anti-D 50 microg/kg. The median platelet count increased from 7 x 10(9) to 31 x 10(9)/L on day 3 (P < 0.01). The median decline in hemoglobin was 1.3 g/dl. Two children experienced minor fever and chills within 24 hr of treatment. Pain at the injection site was common but self-limiting with no effect on activity level. These results suggest subcutaneous anti-D IgG 50 microg/kg as an effective and well-tolerated treatment option in childhood ITP.

[Epidemiology, disease presentation and course of idiopathic thrombocytopenic purpura in Danish children from 1998-2000]. / Epidemiologi, sygdomspraesentation og forløb af idiopatisk trombocytopenisk purpura hos børn i Danmark 1998-2000.

INTRODUCTION: Idopathic Thrombocytopenic Purpura (ITP) is a condition with isolated thrombocytopenia and bleeding symptoms in skin and mucous membranes. It is easy to establish the diagnosis, but treatment is controversial, possibly due to different estimates of the risk for serious bleeding. We present the epidemiology and clinical course of ITP during the first 6 months after diagnosis in Danish children diagnosed from 1998-2000. MATERIALS AND METHODS: The Nordic Society of Paediatric Haematology and Oncology conducted a prospective registration study of children with newly diagnosed ITP from 1998-2000. The study included children < 15 years of age with newly diagnosed, untreated ITP and platelet count (TBC) < 30 x 10(9) /l. Information about bleeding symptoms, TBC, treatment and ITP related episodes was recorded at diagnosis and during the first six months. RESULTS: Of 109 included children 81% had a short symptom history, 46% a preceding viral infection. At diagnosis 62% had TBC < 10 x 10(9) /l, 41% mucosal bleeding, and 72% received medical treatment. Follow-up was available for 91 children. Sixty seven children with acute ITP had TBC < 150 x 10(9) /l for 11 days (median). Twenty four children suffered chronic ITP, 8 of them had persistent TBC < 20 x 10(9) /l. Insidious onset had a 64% predictive value for chronic ITP. Of the 57 registered ITP-related episodes, 45 occurred in chronic cases, 23 of them in six of the children with persistent TBC < 20 x 10(9) /l. CONCLUSION: It is likely that children with newly diagnosed ITP recover quickly, and the risk of serious bleeding is low. A small group of children with persistent severe thrombocytopenia experience significant morbidity.