Management of Relapsed Hairy Cell Leukemia: A Systematic Review of Novel Agents and Targeted Therapies

Raheel Siddiqui, Muhammad Sardar, Moazzam Shahzad, Jemin Jose, Insija Selene, Zunaira Shah, Anum Qureshi, Madeeha Shafqat, Rimsha Kashif, Maheen Ahmad, Alex Mejia-Garcia, Faiz Anwer

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Background: Hairy cell leukemia (HCL) responds well to purine analogs with an overall median relapse free survival of 11-16 years. Most patients can be retreated with the same or a different purine analog however a subset of patients will become resistant or develop cumulative toxicities. Novel agents such as Vemurafenib (BRAF kinase inhibitor), Bendamustine/Rituximab (BR), Moxetumomab pasudotox (anti CD-22 recombinant immunotoxin) and Ibrutinib have emerging roles in patients with relapsed HCL. Methods: Five databases (PubMed, Embase, Cochrane Library, Web of Science and were searched using the following search terms: “hairy cell leukemia” or “leukemia, hairy cell” AND “relapse” or “recurrence”. We included only prospective clinical trials with outcome data. Results: Vemurafenib monotherapy was evaluated in two separate arms of a phase 2 trial. In the US arm (n=24), the ORR was 100% (CR 42%; PR 58%). In the Italian arm (n=26), the ORR was 96% (CR 35%; PR 62%). In a phase 2 study (n=25), the combination of vemurafenib and rituximab showed CR of 100%. The combination of BR achieved an ORR of 100% whereas CR was 50% and 67% at a bendamustine dose of 70mg/m2 (n=6) and 90 mg/m2 (n=6) respectively. In a phase 3 trial, moxetumomab pasudotox (n=80) had an ORR of 75% (CR 41%). Single agent Ibrutinib (n=37) had an ORR of 54%. Therapies were generally well tolerated. Conclusion: Novel agents have good efficacy in HCL in patients with multiple relapses.

Original languageEnglish (US)
Pages (from-to)659-666
Number of pages8
JournalClinical Lymphoma, Myeloma and Leukemia
Issue number10
StatePublished - Oct 2021
Externally publishedYes


  • ibrutinib
  • moxetumomab pasudotox
  • Refractory
  • rituximab
  • vemurafenib

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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