Bone marrow or peripheral blood stem cell transplant

In a prospective randomized trial, 241 previously untreated patients younger than 66 years with advanced-stage disease received induction therapy with a CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisone)-based regimen followed by fludarabine.[24] Complete responders (105 patients) were randomly assigned to undergo autologous stem cell transplant (SCT) or observation, while the other 136 patients were randomly assigned to receive dexamethasone, high-dose cytarabine, and cisplatin reinduction followed by either autologous SCT or fludarabine plus cyclophosphamide (FC). Although the 3-year event-free survival (EFS) favored autologous SCT in complete responders, there was no difference in OS in any of the randomized comparisons.[24][Level of evidence B1] Autologous bone marrow/stem cell transplant is rarely employed for patients with relapsed CLL.

Patients with adverse prognostic factors are very likely to die from CLL. These patients are candidates for clinical trials that employ high-dose chemotherapy and immunotherapy with myeloablative or nonmyeloablative allogeneic peripheral blood SCT.[25-30] Although most patients who attain complete remission after autologous SCT eventually relapse, a survival plateau for allogeneic SCT suggests an additional graft-versus-leukemia effect.[30] A series (NCT00281983) of 90 patients with relapsed or refractory CLL who underwent allogeneic SCT reported a 6-year OS rate of 58% and a 6-year EFS rate of 38%, which included patients with the worst prognostic factors (such as a TP53 pathogenic variant).[31][Level of evidence C2]