[Transplantation of the bone marrow from a HLA-compatible unrelated donor after immunoablative conditioning in children with acquired aplastic anemia unresponsive to combined immunosuppressive therapy: preliminary results].
OBJECTIVE
To analyze the efficiency of transplantation of the bone marrow from a HLA-compatible unrelated donor and continued immunosuppressive therapy (IST) in children with aplastic anemia (AA) unresponsive to 2 courses of IST.


METHODS
The study enrolled 14 children aged 2-16 years (median 9 years). A control group comprised 26 patients in whom IST was continued. The median interval between the diagnosis of AA and transplantation was 26 months (9-156 months). The conditioning regimen consisted of thoracoabdominal irradiation in a dose of 2 Gy, fludarabin (Flu) 100-150 mg/m2, cyclophosphamide (Cy) 100-200 mg/kg, antithymocyte globulin (ATG) in 11 patients and Flu, Cy, and ATG in 3. A graft-versus-host reaction was prevented with mycophenolate mefetil in all the patients, tacrolimus in 11, and cyclosporin A in 3. Donors were compatible for high-resolution typing of 10/10 and 9/10 alleles in 8 and 6 patients, respectively; the source of a transplant was bone marrow in 13 patients and granulocyte colony-stimulating factor-mobilized peripheral blood precursors in one case.


RESULTS
Thirteen patients achieved primary engraftment after single transplantation; one patient did after repeat transplantation. Grades I to II graft-versus-host reaction (GVHR) developed in 9 patients; postengraftment life-threatening infections in 3, extensive chronic GVHR in 2, circumscribed GVHR in 7. All fourteen hemopoietic cell transplant recipients followed for a median 17.5 months (range 1-71 months) were survivors.


CONCLUSIONS
The likelihood of good survival after unrelated transplantations in AA is much higher than that after continued IST: 100% versus 15 +/- 11%.
Novichkova
G A
GA
Maschan
M A
MA
Shipitsyna
I P
IP
Skvortsova
Iu V
IuV
Persiantseva
M I
MI
Lebedeva
L L
LL
Bobrynina
V O
VO
Baĭdil'dina
D D
DD
Goronkova
O V
OV
Solopova
G G
GG
Khachatrian
L A
LA
Petrova
U N
UN
Suntsova
E V
EV
Kalinina
I I
II
Sinitsyna
V V
VV
Skorobogatova
E V
EV
Balashov
D N
DN
Dyshlevaia
Z M
ZM
Shelikhova
L N
LN
Kurnikova
E E
EE
Trakhtman
P E
PE
Maschan
A A
AA
D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy / Q000209:etiology / Q000276:immunology / Q000532:radiotherapy / Q000601:surgery*; D000961:Antilymphocyte Serum / Q000008:administration & dosage / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000008:administration & dosage / Q000627:therapeutic use*; D018572:Disease-Free Survival; D006085:Graft Survival; D006087:Graft vs Host Reaction / Q000276:immunology; D006680:HLA Antigens / Q000235:genetics; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods*; D017211:Treatment Failure
D000961:Antilymphocyte Serum; D006680:HLA Antigens; D007166:Immunosuppressive Agents; D016572:Cyclosporine