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. 2024 May;13(10):e7289.
doi: 10.1002/cam4.7289.

Outcomes of pirtobrutinib for relapsed/refractory mantle cell lymphoma in compassionate use program in Europe

Enver Aydilek  1 , Gerald Wulf  1 , Friedrich Schwarz  1   2 , Ulrike Bacher  3 , Mathias Rummel  4 , Olga Stiefel  5 , Andrea Kerkhoff  6 , Markus Maulhardt  1 , Thomas Melchardt  7 , Thomas Pabst  8 , Georg Lenz  6 , Evgenii Shumilov  6
Affiliations

Outcomes of pirtobrutinib for relapsed/refractory mantle cell lymphoma in compassionate use program in Europe

Enver Aydilek et al. Cancer Med. 2024 May.

Abstract

Background: Mantle cell lymphoma (MCL) is a type of B-cell lymphoma that is currently incurable. Pirtobrutinib shows promising response rates in heavily pretreated MCL patients according to the approval study, but the real-world data are scarce.

Methods: In this study, we retrospectively analyzed the efficacy and safety profile of pirtobrutinib in 10 relapsed/refractory MCL patients from compassionate use program (CUP).

Results: On average, the patients underwent three lines of systemic therapy prior to pirtobrutinib and were predominantly BTKi exposed (9/10). The best overall response rate (BORR) was 67%. In a median follow-up of 8.6 months, the mean duration of response (DOR), progression-free survival (PFS), and overall survival (OS) were not reached. No new safety signals were documented.

Conclusions: In summary, pirtobrutinib represented a safe and effective treatment option in a small real-world population.

Keywords: compassionate use program; mantle cell lymphoma; outcomes; pirtobrutinib.

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Conflict of interest statement

G.L. received research grants not related to this manuscript from AGIOS, AQUINOX, AstraZeneca, Bayer, Celgene, Gilead, Janssen, Morphosys, Novartis, Roche, and Verastem. G.L. received honoraria from ADC Therapeutics, Abbvie, Amgen, AstraZeneca, Bayer, BMS, Celgene, Constellation, Genase, Genmab, Gilead, Hexal/Sandoz, Immagene, Incyte, Janssen, Karyopharm, Lilly, Miltenyi, Morphosys, MSD, NanoString, Novartis, PentixaPharm, Roche, and Sobi.

Figures

FIGURE 1
FIGURE 1
Outcome of treatment from the 10 patients with r/r MCL and pirtobrutinib. (A) Best overall response rates of the patients treated with pirtobrutinib. (B) Duration of response (DOR). (C) Progression‐free survival (PFS). (D) Overall survival (OS). BORR, best overall response rate; CR, complete response; PR, partial response; SD, stable disease; PD, progressive disease. For DOR analysis, only patients showing a response were included. All patients were included for PFS and OS analysis. The survival percentages for 6 and 12 months are given.
See this image and copyright information in PMC

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