Phase I trial of low-dose oral Clofarabine in myelodysplastic syndromes patients who have failed frontline therapy

Venkatesh K. Rudrapatna, Kimberly Morley, Kenneth M. Boucher, Andrew S. Pierson, Christian T. Shull, James P. Kushner, Paul J. Shami

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3 Scopus citations


We investigated protracted low-dose oral Clofarabine for the treatment of myelodysplastic syndromes (MDS). Adults with an International Prognostic Scoring System (IPSS) score of INT-1 or higher who had failed first line therapy were eligible. INT-1 patients had to be transfusion-dependent. We started with oral Clofarabine at 5. mg (fixed dose) daily for 10 consecutive days on a 28-day cycle. Toxicity prompted a modification to 1. mg PO daily for 10 days and then 1. mg PO daily for 7 days. Patients received treatment indefinitely until loss of response or unacceptable toxicity. Nine patients (5 women) were enrolled and evaluable (median age 65 years; range 55-81). A 10-day regimen of oral Clofarabine at 5. mg/day induced Grade IV pancytopenia. A dose of 1. mg/day for 7/28 days was very well tolerated without significant toxicity. Three patients had responses (2 with responses lasting up to 21 and 51 cycles) defined as stable disease in spite of no significant change on bone marrow evaluation. Low-dose oral Clofarabine (1. mg daily for 7/28 days) proved both effective and safe for patients with MDS who had failed prior therapy. This patient population is particularly sensitive to more protracted Clofarabine treatment schedules.

Original languageEnglish (US)
Pages (from-to)835-839
Number of pages5
JournalLeukemia research
Issue number8
StatePublished - Aug 1 2015

Bibliographical note

Funding Information:
This trial was supported by Genzyme .

Publisher Copyright:
© 2015 Elsevier Ltd.


  • Clofarabine
  • Myelodysplasia
  • Myelodysplastic syndrome


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