Faculty, Staff and Student Publications

Language

English

Publication Date

2-1-2026

Journal

Blood Neoplasia

DOI

10.1016/j.bneo.2025.100175

PMID

41536780

PMCID

PMC12796981

PubMedCentral® Posted Date

10-24-2025

PubMedCentral® Full Text Version

Post-print

Abstract

T-cell prolymphocytic leukemia (T-PLL) is a mature T-cell neoplasm with an aggressive clinical course. Overall prognosis is poor, and treatment relies on alemtuzumab because of inadequate response to conventional chemotherapy. Three-quarters of cases harbor activating mutations in the JAK-STAT pathway (JAK1JAK3STAT5BIL2RG). We report safety and efficacy from a phase 1B study evaluating the combination of the JAK1 inhibitor itacitinib with alemtuzumab. Patients (N = 15) were aged >18 years, with treatment-naïve (n = 8) or relapsed/refractory (n = 7) T-PLL with adequate organ function, European Cooperative Oncology Group Performance Status ≤2, and platelet >30 × 103/μL. Cycle 1 included a lead-in phase with itacitinib monotherapy days 1-14. Beginning day 15, patients also received alemtuzumab 30mg IV 3 times weekly for up to 4 (28-day) cycles or until best response. At best response, up to 8 cycles of maintenance with single-agent itacitinib was allowed. Median age was 65 years (range, 39-83). Ten patients (67%) had complex cytogenetics, 11 (73%) had chromosome 14 abnormality, and 13 (87%) were TCL1A positive by fluorescence in situ hybridization. Among frontline patients, overall response rate (ORR) was 88% (complete remission [CR]: 75%), median event-free survival (EFS) and overall survival (OS) were 11.6 and 19.5 months, respectively. Three frontline patients proceeded to allogeneic stem cell transplantation. The ORR in the relapsed/refractory cohort was 57% (CR: 43%), whereas median EFS and OS were 11.1 months. Most (85%) adverse events were grade 1 to 2 and none were attributed to itacitinib. Continued studies evaluating JAK inhibitors in patients with T-PLL are warranted. This trial was registered at www.clinicaltrials.gov as #NCT03989466.

Published Open-Access

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BNEO_NEO-2025-000773-ga1.jpg (374 kB)
Graphical Abstract

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