A National Platform and Scoring System Allocates CAR-T Treatment Slots for Multiple Myeloma to Patients with High Likelihood of Complete Remission

• Published in: Blood 2023-11, Vol.142 (Supplement 1), p.4701-4701
• Main Authors: Driessen, Christoph, Baur, Katharina, Heim, Dominik, Gerull, Sabine, Renner, Christoph, Cairoli, Anne, Pabst, Thomas, Halter, Joerg, Gregor, Michael, Graf, Nicole, Fejzibegovic, Nino, Wolfensberger, Nathan, Simonetta, Federico, Fehr, Martin, Ciliberti, Esteban, Müller, Rouven, Jeker, Barbara
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2023-182628

Introduction Therapy of relapsed and refractory multiple myeloma (MM) with Ide-Cel is superior to alternative treatments, but availability is lower than the number of patients in need. Treatment slots are distributed by the manufacturer per country. Their allocation to individual patients is a medical, logistical and ethical challenge. Ideally, such allocation should be inclusive, fair, transparent, effective, and base on prospectively confirmed objective outcome criteria. Methods Swiss Blood Stem Cell Transplantation and Cellular Therapy (SBST) has established the national SBST-CAR-T MM board for allocation of Ide-Cel treatment slots. Allocation process and triage criteria were developed by clinical experts in exchange with the ethical board of the Swiss academy for medical sciences and health insurers, approved by all JACIE-accredited national centers for cellular therapies, and agreed that Ide-Cel therapy in Switzerland is only available through this allocation system. Patients are registered in parallel with request for insurance coverage in one key document. Patients had to meet qualifying approval criteria for Ide-Cel in Switzerland (relapsed and refractory MM, >2 prior therapy lines, triple-class-exposed). Reaching CR after Ide-Cel is associated with superior PFS. We therefore developed an algorithm to select patients with high likelihood for CR. Patient factors negatively associated with CR derived from the KarMMa trial were complemented by factors prospectively associated with inferior overall survival after CAR-T in malignant lymphoma. High tumor burden (bone marrow infiltration > 50% or high tumor load by PET-CT), inflammatory activity (increasede ferritin or D-dimers), ECOG patient status and comorbidities were used as negative predictive factors and combined to a sum score of 0-4. All registered patients are presented at the monthly virtual meeting and ranked according to this score. Ranking is refined based on availability of alternative treatment options, resulting in a final national ranking agreed by all board members. Because no patients with severe comorbidities were registered, this dimension was later excluded. Results Between May 2022 and April 2023 all 48 Ide-Cel regular treatment slots in Switzerland were allocated in 12 virtual meetings lasting median 55 minutes (25-70) with the participation of median 7 centers (6-8), 8 physicians (6-15), discussing in total 72 patients (median 5.5 per meeting, range 4-10) to distribute 4 slots mo