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Blood, Vol. 93 No. 12 (June 15), 1999:
pp. 4125-4130
Cladribine Activity in Adult Langerhans-Cell Histiocytosis
Alan Saven and
Carol Burian
From the Division of Hematology/Oncology, Ida M. and Cecil H. Green
Cancer Center, Scripps Clinic, La Jolla, CA.
Langerhans-cell histiocytosis (LCH) results from the accumulation of
tissue histiocytes derived from the same progenitor cells as monocytes.
Because cladribine is potently toxic to monocytes, we conducted a phase
II trial of cladribine. Cladribine was administered to 13 LCH patients
at 0.14 mg/kg per day by 2-hour intravenous infusion for 5 consecutive
days, every 4 weeks for a maximum of six courses. Median age was 42 years (range, 19 to 72) and median pretreatment disease duration was 99 months (range, 6 to 252). One patient was untreated, one had received
prior prednisone only, one prior radiation only, six prior radiation
and chemotherapy, and four prior surgery, radiation, and chemotherapy.
Seven patients had cutaneous involvement, six multifocal osseous, six
pulmonary, two each with soft tissue and nodal involvement, and four
had diabetes insipidus. Of 13 patients, 12 were evaluable for response and all for toxicity. After a median of three courses (range, 1 to 6),
seven (58%) patients achieved complete responses (two pathologic and
five clinical) and two (17%) patients achieved partial responses;
overall response rate, 75%. Median response follow-up duration was 33 months (range, 1 to 65). Seven patients experienced grade 3 to 4 neutropenia. Only one patient had a documented infection, dermatomal
herpes zoster. At a median follow-up of 42 months (range, 5 to 76), 12 patients remain alive and one patient has died. Thus, cladribine has
major activity in adult LCH and warrants further investigation in both
pediatric and adult LCH as a single agent and in combination with other drugs.

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