Phase 1 study of pomalidomide and dexamethasone for relapsed/refractory primary CNS or vitreoretinal lymphoma Journal Article


Authors: Tun, H. W.; Johnston, P. B.; DeAngelis, L. M.; Atherton, P. J.; Pederson, L. D.; Koenig, P. A.; Reeder, C. B.; Omuro, A. M. P.; Schiff, D.; O'Neill, B.; Pulido, J.; Jaeckle, K. A.; Grommes, C.; Witzig, T. E.
Article Title: Phase 1 study of pomalidomide and dexamethasone for relapsed/refractory primary CNS or vitreoretinal lymphoma
Abstract: The combination of pomalidomide (POM) and dexamethasone (DEX) was evaluated for relapsed/refractory primary central nervous system lymphoma (PCNSL) and primary vitreoretinal lymphoma (PVRL) to determine the maximal tolerated dose (MTD) of POM as the primary objective, and overall response rate (ORR), progression-free survival (PFS), and safety profile as secondary objectives. A cohorts-of-3 study design was used with a dose-escalation schedule consisting of POM (3, 5, 7, or 10 mg) orally daily for 21 days every 28 days and DEX 40 mg orally every week. After 2 cycles, POM was continued alone until disease progression, intolerance, or subject withdrawal. Following MTD determination, the MTD cohort was expanded. Twenty-five of 29 patients with the median of 3 prior treatments were eligible for assessment as per international PCNSL collaborative group criteria. The MTD of POM was 5 mg daily for 21 days every 28 days. Whole-study ORR was 48% (12 of 25; 95% confidence interval [CI], 27.8%, 68.7%) with 6 complete response (CR), 2 complete response, unconfirmed (CRu), and 4 partial response (PR). MTD cohort ORR was 50% (8 of 16; 95% CI, 24.7%, 75.4%) with 5 CR, 1 CRu, and 2 PR. Median PFS was 5.3 months (whole study) and 9 months (for responders). One patient had pseudoprogression. Grade 3/4 hematologic toxicities included neutropenia (21%), anemia (8%), and thrombocytopenia (8%). Grade 3/4 nonhematologic toxicities included lung infection (12%), sepsis (4%), fatigue (8%), syncope (4%), dyspnea (4%), hypoxia (4%), respiratory failure (8%), and rash (4%). POM/DEX treatment is feasible with significant therapeutic activity against relapsed/refractory PCNSL and PVRL. This trial was registered at www.clinicaltrials.gov as #NCT01722305. © 2018 by The American Society of Hematology.
Journal Title: Blood
Volume: 132
Issue: 21
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2018-11-22
Start Page: 2240
End Page: 2248
Language: English
DOI: 10.1182/blood-2018-02-835496
PROVIDER: scopus c2 - 30262659
PMCID: PMC6265643
PUBMED: 30262659
DOI/URL:
Notes: Blood -- Export Date: 2 January 2019 -- Article -- Source: Scopus C2 - 30262659
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MSK Authors
  1. Antonio Marcilio Padula Omuro
    176 Omuro
  2. Christian Grommes
    52 Grommes