改善欧洲儿童 B-ALL 获得 CD19 CAR-T 细胞治疗可及性的基线可及性与多层次障碍全景评估（IMPACT 研究）

Chimeric antigen receptor T-cell therapy (CAR-T) has revolutionized the treatment of B-cell precursor ALL (B-ALL), but its global availability is limited. This study assessed current access and barriers to CAR-T CD19 therapy for children across Europe.

A country questionnaire developed by the European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party, St Jude Children's Research Hospital, and IBFM assessed current access to advanced therapies for B-ALL in Europe using Qualtrics software.

Data from 36 WHO-defined European countries (27 high-income, nine upper-middle-income) observed a median of five pediatric hematology-oncology (PHO) centers per country (0.56 PHO centers/1 million inhabitants, range, 0.05-1.83). Hematopoietic stem-cell transplantation (HSCT) facilities were available in 89% of countries (32/36). CAR-T CD19 therapy was available in 72% of countries; however, 25/36 (69%) countries lacked clinical trials or international collaborations for pediatric CAR-T CD19 therapy. Most countries accepted foreign patients, but referrals remained limited, with 1-2 foreign patients treated annually per country. Eighteen countries expressed interest in a referral network, but only six had established mechanisms for domestic or international referrals.

Substantial disparities exist in access to advanced therapies for pediatric B-ALL across Europe. Although CAR-T CD19 therapy is available in most countries, gaps in clinical trials, collaborations, and referral systems limit equitable access. Efforts to improve infrastructure and establish referral networks are essential to enhance care for patients with pediatric B-ALL.