Allogeneic hematopoietic stem cell transplantation for patients with hemophagocytic syndrome (HPS) in Japan.
Seventeen cases (age at onset, 1 month to 18 years; M/F, 9/8) of hemophagocytic syndrome which received allogeneic hematopoietic stem cell transplantation (SCT) in Japan during the period 1988-1998 are reported. The patients consisted of six familial inheritance-proven erythrophagocytic lymphohistiocytosis (FEL), five familial inheritance-unknown and infective agents-unknown HLH (of which two were highly likely to have been FEL with characteristic CNS signs), and six aggressive Epstein-Barr virus (EBV)-related HLH (of which two were natural killer cell-type large granular leukemia/lymphoma-associated hemophagocytic syndrome, EBV-NK-LGLL-HPS). All cases were treated intensively with immuno-chemotherapy, or with chemotherapy before SCT. As sources of SCT, 12 cases received bone marrow cells (sibling six, father one, URD five), two cord blood, two purified CD34-positive cells, and one PBSC. SCTs were successful in all 17 cases, apart from one receiving CD34-positive SCT. Following SCT, four patients relapsed and five died with a median follow-up of 23 months. Among the relapsed cases, the two EBV-NK-LGLL-HPS previously published as successfully transplanted were included. Among the fatal cases, three patients died from relapsed active disease and the remaining two from fatal post-SCT EBV-positive T cell lymphoma and extensive chronic GVHD, respectively. As of the end of September 1998, 10 patients are alive without disease for 3.5 months to 147 months, while two post-SCT patients are still having therapy for residual/recurrent disease. The Kaplan-Meier analysis showed a 2-year event-free survival after SCT as 54.0+/-13.0%.
Imashuku
S
S
Kyoto Prefectural University of Medicine, Japan.
Hibi
S
S
Todo
S
S
Sako
M
M
Inoue
M
M
Kawa
K
K
Koike
K
K
Iwai
A
A
Tsuchiya
S
S
Akiyama
Y
Y
Kotani
T
T
Kawamura
Y
Y
Hirosawa
M
M
Hasegawa
D
D
Kosaka
Y
Y
Yamaguchi
H
H
Ishii
E
E
Kato
K
K
Ishii
M
M
Kigasawa
H
H
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D015616:Histiocytosis, Non-Langerhans-Cell / Q000453:epidemiology / Q000628:therapy*; D006801:Humans; D007223:Infant; D007564:Japan / Q000453:epidemiology; D008297:Male; D013997:Time Factors; D014019:Tissue Donors