[Treatment of acquired severe aplastic anemia in pediatric patients with immunosuppression and allogeneic stem cell hematopoietic transplant].
BACKGROUND
Severe acquired aplastic anemia (SAA) is an uncommon disease of childhood. Patients with SAA receive supportive care with transfusions and timely treatment of opportunistic infections, along with specific therapies, which may be allogenic stem cell transplantation (SCT) from a matched sibling or immunosuppressive therapy (IT).


OBJECTIVE
To report the experience in the management of SAA.


METHODS
Twenty five children with acquired SAA were treated from July 1992 to September 2005. Patients with full matched sibling donors received allogenic SCT after conditioning with a cyclophosphamide containing regimen. The other patients received immune suppression with cyclosporine plus methylprednisolone (n= 18) plus ATG (n=17). All received supportive care until recovery of hematopoietic function. Those who had severe opportunistic infections at diagnosis or did not respond to two cycles of ATG were evaluated for unrelated donor SCT.


RESULTS
Seven patients received sibling donor SCT and 18 IT, which was repeated in six. Three patients received mismatched related (1) or unrelated (2) SCT. Nineteen patients survived with a median follow up time of 4 years, 14 with full hematologic recovery. Six patients died: four due to infections after IT or SCT, one due to intracranial hemorrhage and one with secondary myelodysplasia 12 years after IT.


CONCLUSIONS
Most children with SAA can be treated successfully with sibling donor SCT or IT. Patients without a histocompatible sibling who fail to respond to IS have a worse prognosis.
Barriga C
Francisco
F
Departamento de Pediatría, Pontificia Universidad Católica de Chile, Santiago, Chile. fbarriga@med.puc.cl
Wietstruck P
Angélica
A
Becker K
Anna
A
Zúñiga C
Pamela
P
Besa De C
Pelayo
P
Alvarez Z
Manuel
M
Ocqueteau T
Mauricio
M
Bertín C
Pablo
P
D000293:Adolescent; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008775:Methylprednisolone / Q000627:therapeutic use; D011379:Prognosis; D012307:Risk Factors; D012720:Severity of Illness Index; D014184:Transplantation, Homologous; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D016572:Cyclosporine; D003520:Cyclophosphamide; D008775:Methylprednisolone