Publications by authors named "Y Reguerre"

SACHA-France (NCT04477681) is a prospective real-world study that collects clinical safety and efficacy data of novel anticancer therapies prescribed off-label or on compassionate use to patients <25 years. From March 2020 until February 2024, 640 patients with solid tumors or lymphomas were included, with 176 (28%) reported objective tumor responses. Centralized medical monitoring of local radiological/functional imaging reports by the SACHA coordinating investigator led to response modification in 45 out of 176 cases (26%), highlighting the relevance of the medical review of study data.

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Acute leukemias of ambiguous lineage (ALAL) represent between 3 and 5% of childhood AL. This term encompasses many subtypes of AL that have been defined according to the immunophenotypic profile based on the expression of various lineage markers. This classification has been modified and enriched during the last decade thanks to the improvement of molecular biology techniques, which have led to reconsider the ontogenic proximity existing between certain forms of ALAL.

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Article Synopsis
  • Hematological involvement (HI) is a serious condition associated with Langerhans cell histiocytosis (LCH), and is defined by low blood cell counts indicating potential life-threatening complications.
  • A study of 2,313 children with LCH revealed that about 331 developed HI, with a distinction between mild (MHI) and severe HI (SHI), each showing different health outcomes and survival rates over 10 years.
  • Recent advances in treatment have improved mortality rates, particularly with combination therapies and targeted treatments, sparking discussions about refining HI definitions to better inform treatment strategies.
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Article Synopsis
  • - Gastrointestinal stromal tumours (GIST) are digestive tract tumors primarily affecting adults, with limited management guidelines for pediatric cases.
  • - An updated literature review reveals that GIST in children often links to syndromic or familial contexts, with over 170 documented cases, primarily involving genetic alterations in Succinate Dehydrogenase (SDH).
  • - Management recommendations for pediatric GIST are similar to those for adults, but specific biological differences necessitate tailored therapeutic approaches, prompting international collaborations to enhance understanding of this rare condition.
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Purpose: Within the Paediatric Rare Tumours Network-European Registry (PARTNER) project, we aimed to evaluate the situation on the registration and management of paediatric patients affected by very rare tumours (VRT) in the European low health expenditure average rates (LHEAR) countries.

Methods: A survey regarding infrastructure, organisation, and clinical decision-making information on VRT was designed. This survey was distributed to the representatives of LHEAR countries involved in the activities of the PARTNER Work Package 7.

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