Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia

Robert J. Kreitman, Claire Dearden, Pier Luigi Zinzani, Julio Delgado, Lionel Karlin, Tadeusz Robak, Douglas E. Gladstone, Philipp le Coutre, Sascha Dietrich, Mirjana Gotic, Loree Larratt, Fritz Offner, Gary Schiller, Ronan Swords, Larry Bacon, Monica Bocchia, Krimo Bouabdallah, Dimitri A. Breems, Agostino Cortelezzi, Shira DinnerMichael Doubek, Bjorn Tore Gjertsen, Marco Gobbi, Andrzej Hellmann, Stephane Lepretre, Frederic Maloisel, Farhad Ravandi, Philippe Rousselot, Mathias Rummel, Tanya Siddiqi, Tamar Tadmor, Xavier Troussard, Cecilia Arana Yi, Giuseppe Saglio, Gail J. Roboz, Kemal Balic, Nathan Standifer, Peng He, Shannon Marshall, Wyndham Wilson, Ira Pastan, Nai Shun Yao, Francis Giles

Research output: Contribution to journalArticlepeer-review

183 Scopus citations


This is a pivotal, multicenter, open-label study of moxetumomab pasudotox, a recombinant CD22-targeting immunotoxin, in hairy cell leukemia (HCL), a rare B cell malignancy with high CD22 expression. The study enrolled patients with relapsed/refractory HCL who had ≥2 prior systemic therapies, including ≥1 purine nucleoside analog. Patients received moxetumomab pasudotox 40 µg/kg intravenously on days 1, 3, and 5 every 28 days for ≤6 cycles. Blinded independent central review determined disease response and minimal residual disease (MRD) status. Among 80 patients (79% males; median age, 60.0 years), durable complete response (CR) rate was 30%, CR rate was 41%, and objective response rate (CR and partial response) was 75%; 64 patients (80%) achieved hematologic remission. Among complete responders, 27 (85%) achieved MRD negativity by immunohistochemistry. The most frequent adverse events (AEs) were peripheral edema (39%), nausea (35%), fatigue (34%), and headache (33%). Treatment-related serious AEs of hemolytic uremic syndrome (7.5%) and capillary leak syndrome (5%) were reversible and generally manageable with supportive care and treatment discontinuation (6 patients; 7.5%). Moxetumomab pasudotox treatment achieved a high rate of independently assessed durable response and MRD eradication in heavily pretreated patients with HCL, with acceptable tolerability.

Original languageEnglish (US)
Pages (from-to)1768-1777
Number of pages10
Issue number8
StatePublished - Aug 1 2018
Externally publishedYes

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


Dive into the research topics of 'Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia'. Together they form a unique fingerprint.

Cite this