Background: Stem-cell transplantation is a reasonable therapeutic approach for younger patients with high-risk CLL.
Patients and methods: Twelve patients (seven males; median age 47 years, range 29-51) with high-risk CLL underwent transplantation (allo, n = 7; auto, n = 5). The conditioning regimen consisted of cyclophosphamide and total body irradiation in 11 patients, and BEAC in the remaining one. Minimal residual disease (MRD) was assessed by cytofluorometry and PCR.
Results: All 11 evaluable patients engrafted. Of the seven allografted patients, two died of treatment-related causes; three patients developed acute GVHD. No transplant-related mortality was observed in autografted patients. After transplantation, 10 of 11 patients evaluable for response achieved CR (91%; 95% CI 59%-100%) which was molecular in nine patients (82%; 95% CI 48%-98%). One patient in CR but MRD+ relapsed nine months after transplantation and died. Seven patients remain in molecular CR for a median of 16 months (range 1-58). Estimated actuarial survival and disease-free survival at two years is 81% (95% CI 43%-100%) and 71% (95% CI 43%-99%), respectively. Relapse risk at two years is 12.5% (95% CI 0%-35.5%).
Conclusions: Patients with high-risk CLL can achieve long-lasting molecular CR after SCT. The role of transplants in CLL management deserves investigation in controlled trials.