STEROID-REFRACTORY CUTANEOUS GRAFT-VERSUS-HOST DISEASE AFTER TRANSPLANTATION OF HAPLOIDENTICAL PARENTAL CD34(-SYNDROME AND RECURRENT ACUTE-LEUKEMIA() CELLS IN CHILDREN WITH DOWNS)

Purpose: This study evaluated the feasibility of performing haploident ical CD34(+) selected transplants for children with Down's syndrome (D S) and recurrent leukemia. Patients and Methods: Within a cohort of 15 children, two patients had DS. Transplantation of CD34(+) cells from haploidentical parents was performed after the children were condition ed with fractionated total body irradiation, cyclophosphamide, and ant ithymocyte globulin (ATG). Graft-versus-host disease (GVHD) prophylaxi s consisted of cyclosporine and a short course of methotrexate. Result s: The preparative regimen was well tolerated, and engraftment of poly morphonuclear cells and platelets took place promptly (by day 20) in b oth patients with DS. However, both patients with DS experienced sever e grade IV GVHD that was limited to the skin and was refractory to sal vage with high-dose methylprednisolone therapy. In one patient, GVHD r esponded to second-line salvage therapy with ATG, but the patient died on day 234 from leukemic relapse. The second patient had GVHD that di d not respond to ATG and died of multisystem organ failure and refract ory GVHD on day 44. Two of two DS patients had steroid refractory seve re acute GVHD of the skin, while only one of 11 evaluated and identica lly treated non-DS patients had severe GVHD (p < 0.05). Conclusion: Th ese observations in patients who underwent mismatched bone marrow tran splantation suggests that patients with DS have an increased risk of s evere acute GVHD of the skin in this context.