Stem cell transplantation with reduced-intensity conditioning for hemophagocytic lymphohistiocytosis.
Allogeneic stem cell transplantation (SCT) is curative for hemophagocytic lymphohistiocytosis (HLH). However, patients frequently have significant morbidity before transplantation and there is high transplant-related mortality (TRM). Because first-degree HLH is caused by immune dysregulation, a reduced-intensity conditioned (RIC) regimen might be sufficient for cure while decreasing the TRM. Twelve patients with HLH underwent RIC SCT from a matched family/unrelated or haploidentical donor. Eleven were conditioned with fludarabine/melphalan with additional busulphan for haploidentical grafts. One received fludarabine and 2-Gy total body irradiation (TBI). All patients showed engraftment at a median of 14 days. Nine of 12 (75%) are alive and in complete remission (CR) a median of 30 months (range, 9-73 months) after SCT. Two patients died from pneumonitis and one from hepatic rupture. Four patients developed acute graft-versus-host disease (GVHD) and 3 have chronic GVHD. Three of 9 survivors have mixed chimerism but remain free of disease. In summary, RIC compares favorably to conventional SCT with long-term disease control in surviving patients despite a significant incidence of mixed chimerism.
Cooper
Nichola
N
Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom.
Rao
Kanchan
K
Gilmour
Kimberly
K
Hadad
Lema
L
Adams
Stuart
S
Cale
Cathy
C
Davies
Graham
G
Webb
David
D
Veys
Paul
P
Amrolia
Persis
P
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy / Q000401:mortality; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality; D006801:Humans; D007223:Infant; D008107:Liver Diseases / Q000209:etiology / Q000401:mortality; D051359:Lymphohistiocytosis, Hemophagocytic / Q000150:complications / Q000401:mortality / Q000628:therapy*; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage*; D011014:Pneumonia / Q000209:etiology / Q000401:mortality; D012189:Retrospective Studies; D012422:Rupture, Spontaneous / Q000209:etiology / Q000401:mortality; D033581:Stem Cell Transplantation; D018183:Transplantation Chimera; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D014916:Whole-Body Irradiation
D019653:Myeloablative Agonists