Development of the SIOPE DIPG network, registry and imaging repository: a collaborative effort to optimize research into a rare and lethal disease.

Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly childhood malignancy. After 40 years of mostly single-center, often non-randomized trials with variable patient inclusions, there has been no improvement in survival. It is therefore time for international collaboration in DIPG research, to provide new hope for children, parents and medical professionals fighting DIPG. In a first step towards collaboration, in 2011, a network of biologists and clinicians working in the field of DIPG was established within the European Society for Paediatric Oncology (SIOPE) Brain Tumour Group: the SIOPE DIPG Network. By bringing together biomedical professionals and parents as patient representatives, several collaborative DIPG-related projects have been realized. With help from experts in the fields of information technology, and legal advisors, an international, web-based comprehensive database was developed, The SIOPE DIPG Registry and Imaging Repository, to centrally collect data of DIPG patients. As for April 2016, clinical data as well as MR-scans of 694 patients have been entered into the SIOPE DIPG Registry/Imaging Repository. The median progression free survival is 6.0 months (95% Confidence Interval (CI) 5.6-6.4 months) and the median overall survival is 11.0 months (95% CI 10.5-11.5 months). At two and five years post-diagnosis, 10 and 2% of patients are alive, respectively. The establishment of the SIOPE DIPG Network and SIOPE DIPG Registry means a paradigm shift towards collaborative research into DIPG. This is seen as an essential first step towards understanding the disease, improving care and (ultimately) cure for children with DIPG.

Value of routine bone marrow examination in pediatric acute myeloid leukemia (AML): a study of the Dutch Childhood Oncology Group (DCOG).

BACKGROUND: The outcome of the treatment of pediatric acute myeloid leukemia (AML) is still disappointing, due to relatively high treatment-related mortality and relapse rates (30-40%). Past treatment protocols have called for routine screening via bone marrow aspiration (BMA) after achievement of first complete remission (CR1) to detect relapse at an early stage. However, supporting evidence for this policy is lacking for non-FAB type-M3 patients. PROCEDURE: We therefore retrospectively studied the clinical relevance of routine BMA in an unselected cohort of all pediatric AML patients in the Netherlands. RESULTS: Of 440 patients, data for 349 patients, of whom 148 suffered bone marrow relapse (BM-relapse), could be analyzed. A total of 1,790 BMAs had been performed, 1,648 (92%) routinely, and 142 (8%) on indication when a relapse was suspected. Forty routine BMAs showed BM-relapse (2% of all routine BMAs), while as many as 108 (76%) hematological relapses were confirmed by BMA on indication (P < 0.001). Therefore, 1 in 41 routine BMAs, as opposed to 1 in 1.3 BMAs performed on indication, detected a BM-relapse. CONCLUSIONS: Routine BMA after CR1 did not significantly contribute to early detection of relapsed AML. These results suggest that BMA after achievement of CR1 should only be performed on indication or within a clinical research setting. Pediatr Blood Cancer 2012; 59: 1239-1244. © 2012 Wiley Periodicals, Inc.