[Results of immunosupressive therapy in children with severe aplastic anaemia. Report of the Polish Paediatric Haematology Group].
BACKGROUND
Bone marrow transplantation from HLA identical family donors is the treatment of choice for children with severe aplastic anaemia (SAA). When there is no donor available, combined immunosuppressive therapy is given.


OBJECTIVE
evaluation of results of immunosupressive therapy in children with severe aplastic anaemia.


METHODS
SAA was diagnosed in 105 children (42 girls, 73 boys), aged 2-18 years, in the eleven haematological centres in Poland, between 1993-2007. All patients received the Severe Aplastic Anaemia Working Party of the EBMT protocol which included: antilymphocyte globulin or antithymocyte globulin, cyclosporin A, prednisolone. Granulocyto- or granulocytomacrophagic-cell stimulation factor was additionally administered during deep neutropenia. Haematological response was evaluated on day 84 or 112 and 180 of the therapy.


RESULTS
complete remission occurred in 53 patients (51.5%), partial remission in 27 (24.7%), no response was obtained in 25 children (23.8%) on day 180, of the therapy. Period of observation was from 12 months to 12.5 years. During this time relapse occurred in 10 patients (9.5%). We observed 22 deaths: 8 early, during the first 3 months of IS and 14 after the first 3 months of immunosuppresive therapy (IS). At present 70 children (66.6%) are in first remission with lasts from 12 months to 12.5 years. The survival at 12.5-years is 78.6%. During the 12.5 years of follow-up we had two cases with a late clonal complication (PNH and MDS). Transformation to acute nonlymphoblastic leukaemia was observed in two of our patients.


CONCLUSIONS
1. Immunosuppresive therapy (IS) in children with SAA, without bone marrow family donors, is more effective after introduction of combined IS (12.5 years survival in this study was 80% for children with very severe aplastic anaemia (v SAA). 2. In our studies among the children followed up after IS therapy, there were: 1 case of periodic nocturnal haemoglobinuria (PNH), 1 case of myelodysplastic syndrome (MDS) and 2 cases of myeloid leukaemia (probability of incidence was 3.8%).
Pawelec
Katarzyna
K
Katedra i Klinika Pediatrii, Hematologii i Onkologii WUM SPDSK, ul. Marszałkowska 24, 00-576 Warszawa, Poland. katpawelec@poczta.onet.pl
Matysiak
Michał
M
Niewiadomska
Edyta
E
Rokicka-Milewska
Roma
R
Kowalczyk
Jerzy
J
Stefaniak
Jolanta
J
Balwierz
Walentyna
W
Załecka-Czerpko
Ewa
E
Chybicka
Alicja
A
Szmyd
Krzysztof
K
Sońta-Jakimczyk
Danuta
D
Bubała
Halina
H
Krauze
Agnieszka
A
Wysocki
Mariusz
M
Kurylak
Andrzej
A
Wachowiak
Jacek
J
Grund
Grzegorz
G
Młynarski
Wojciech
W
Bulas
Monika
M
Krawczuk-Rybak
Maryna
M
Leszczyńska
Elzbieta
E
Urasiński
Tomasz
T
Peregud-Pogorzelski
Jarosław
J
Balcerska
Anna
A
Włazłowski
Marek
M
D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000401:mortality; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D011239:Prednisolone / Q000627:therapeutic use; D012074:Remission Induction; D016019:Survival Analysis; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D011239:Prednisolone