Correspondence

Treatment of Systemic Mast-Cell Disease with Cladribine

N Engl J Med 2001; 344:307-309January 25, 2001

Article

To the Editor:

Cladribine (2-chlorodeoxyadenosine) is a purine nucleoside analogue with activity against various hematologic cancers.1 Because mast cells are derived from hematopoietic stem cells,2 we evaluated the therapeutic activity of cladribine in a patient with interferon alfa–resistant systemic mast-cell disease.

A 57-year-old man was given a diagnosis of systemic mast-cell disease in 1975. At that time, he presented with back pain due to multiple compression fractures of the vertebrae and urticaria pigmentosa. After treatment with spinal irradiation, the course of the patient's illness was indolent, although he continued to have skin lesions. In 1994, he had an episode of flushing, hypotension, and syncope. Treatment was started with histamine H₁- and H₂-receptor blockers and subcutaneous interferon alfa (3 million units administered three times a week). During 14 months of treatment with interferon alfa, the patient had severe side effects without any evidence of improvement in the skin lesions or flushing. Bone marrow examination performed both before and after treatment with interferon alfa revealed extensive mast-cell infiltration without any change in the interim (Figure 1AFigure 1Bone Marrow–Biopsy Specimens Obtained before and after Treatment with Cladribine in a 57-Year-Old Man with Systemic Mast-Cell Disease. and Figure 1B).

The patient was subsequently treated with intravenous cladribine (a two-hour infusion given at a dose of 0.13 mg per kilogram of body weight per day for five days) for a total of six cycles. The interval between each five-day cycle was six months.

After four cycles of treatment with cladribine, the skin lesions had completely resolved. A follow-up bone marrow examination showed a marked reduction in mast-cell infiltration. In November 1999 (one year after the last cycle of cladribine had been administered), the patient continued to be free of all manifestations of disease, including skin lesions, and a bone marrow biopsy revealed only minimal residual disease (Figure 1C and Figure 1D).

Our experience supports the consideration of treatment with cladribine in an adult with systemic mast-cell disease that is aggressive and unresponsive to usual therapy.

Ayalew Tefferi, M.D.
Chin-Yang Li, M.D.
Joseph H. Butterfield, M.D.
H. Clark Hoagland, M.D.
Mayo Clinic, Rochester, MN 55905

2 References

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