[Treatment of refractory rheumatism among preschool children with autologous peripheral blood hematopoietic stem cell transplantation].
OBJECTIVE
To investigate the feasibility and safety of autologous peripheral blood hematopoietic stem cell transplantation (auto-PBHSCT) and its therapeutic effect on refractory rheumatism among preschool children.


METHODS
Three boys with juvenile rheumatoid arthritis (JRA), juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM) respectively, 3 to 6 years old with the mean age of 5 years with 3.5 to 22 months course of disease with 14 months on average, received auto-PBHSCT. Their conditions were so severe that conventional therapy failed to control the diseases. The changes of both clinical manifestations and immunologic indexes were observed before and after transplantation with long term following up at specialty clinic of rheumatism.


RESULTS
The time when neutrophil count >or= 0.5 x 10(9)/L in the 3 children was days +9, +13 and +11 respectively, that of platelet count >or= 20 x 10(9)/L was days +14, +18 and +13 respectively. The cellular immune function remained abnormal with CD4 cells at a low level and CD4/CD8 being inverted. As to the JDM child, the skin rash had disappeared and his muscle tone was improved to grade 5 within one month after the transplantation. The EMG and serum creatase level returned to normal and muscle MRI findings were improved greatly within 2 months after the transplantation. As to the JSLE child, skin rash and proteinuria had disappeared, MRI of brain showed that the pathological changes had been absorbed and EEG returned to normal 3 months after the transplantation, all the autoantibodies turned to negative within 8 months after transplantation. As to the JRA child, the arthritis had been improved remarkably within 3 weeks after auto-PBHSCT. There was no swelling of joints nor movement limitation 3 months post transplantation. The steroids and immunosuppressive drugs were discontinued post transplantation. Cushing syndrome disappeared. Their body heights increased by 10 to 15 cm in the past 18 months, and they all returned to school. There was no relapse during follow-up periods of 25 - 27 months.


CONCLUSIONS
The therapy with auto-PBHSCT for refractory rheumatism among preschool children was remarkably effective in a short-term, yet the safety and long-term effect still need to be further studied.
Wu
Feng-qi
FQ
Department of Pediatric Rheumatology, Capital Institute of Pediatrics, Beijing 100020, China.
Luan
Zuo
Z
Lai
Jian-ming
JM
Tang
Xiang-feng
XF
Lu
Jie
J
Liu
Zhe-wei
ZW
Wang
Tian-you
TY
D002648:Child; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation; D012216:Rheumatic Diseases / Q000628:therapy*; D014182:Transplantation, Autologous; D016896:Treatment Outcome