Minimal residual hairy cell leukemia eradication with moxetumomab pasudotox: Phase 1 results and long-term follow-up Journal Article


Authors: Kreitman, R. J.; Tallman, M. S.; Robak, T.; Coutre, S.; Wilson, W. H.; Stetler-Stevenson, M.; FitzGerald, D. J.; Santiago, L.; Gao, G.; Lanasa, M. C.; Pastan, I.
Article Title: Minimal residual hairy cell leukemia eradication with moxetumomab pasudotox: Phase 1 results and long-term follow-up
Abstract: Anti-CD22 moxetumomab pasudotox achieved 46% complete remissions (CRs) in previously reported phase 1 testing in relapsed/refractory hairy cell leukemia (HCL; n 5 28). The importance of minimal residual disease (MRD) after CR in HCL is unknown. A 21-patient extension cohort received 50 mg/kg every other day for 3 doses in 4-week cycles. These patients plus 12 previously reported at this upper dose level received 143 cycles without dose-limiting toxicity. The combined 33-patient cohort achieved 64% CR and 88% overall response rates, with median CR duration of 42.4 months. Of 32 50-mg/kg patients evaluable for MRD by bone marrow aspirate flow cytometry (most stringent assessment), median CR duration was 13.5 (4.9-42.4) months in 9 MRD-positive CRs vs 42.1 (24.0-69.2) months in 11 MRD-negative CRs (P < .001). Among MRD-negative CRs, 10 patients had ongoing CR, 9 without MRD, at end of study. To our knowledge, moxetumomab pasudotox is the only nonchemotherapy regimen that can eliminate MRD in a significant percentage of HCL patients, to enhance CR duration. Repeated dosing, despite early neutralizing antibodies, increased active drug levels without detectable toxicity from immunogenicity. The activity and safety profiles of moxetumomab pasudotox support ongoing phase 3 testing in HCL. Copyright 2011 by The American Society of Hematology.
Keywords: adult; clinical article; controlled study; treatment outcome; aged; cancer combination chemotherapy; treatment duration; rituximab; flow cytometry; follow up; cd8+ t lymphocyte; tumor volume; cohort analysis; sex ratio; drug distribution; bone marrow biopsy; minimal residual disease; cd4+ t lymphocyte; long term care; drug clearance; disease duration; phase 1 clinical trial; drug half life; disease exacerbation; pentostatin; hairy cell leukemia; spleen size; tumor immunogenicity; moxetumomab pasudotox; human; male; female; priority journal; article; disease eradication; minimal residual hairy cell leukemia
Journal Title: Blood
Volume: 131
Issue: 21
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2018-05-24
Start Page: 2331
End Page: 2334
Language: English
DOI: 10.1182/blood-2017-09-803072
PROVIDER: scopus
PMCID: PMC5969375
PUBMED: 29487070
DOI/URL:
Notes: Article -- Export Date: 2 July 2018 -- Source: Scopus
Altmetric Score
MSK Authors
  1. Martin Stuart Tallman
    443 Tallman