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


OBJECTIVE
Evaluation of results of immunosuppressive therapy in children with severe aplastic anaemia.


METHODS
SAA was diagnosed in 85 children (31 girls, 54 boys) aged 2-17.5 years in the eleven centres of the Polish Paediatric Leukaemia and Lymphoma Study Group (PPLLSG) in Poland between 1993-2003 years. All patients received protocol of the Severe Aplastic Anaemia Working Party of the Europe Bone Marrow Transplant (EBMT): antilymphocyte globulin or antithymocyte globulin, cyclosporin A, prednisolone and granulocyto- or granulocyto-macrophagic-cell stimulation factor was additionally administered during deep neutropenia. Haematological response was evaluated on day 84, 112 or 180 of the therapy.


RESULTS
complete remission occurred in 43 patients (50.5%), partial remission in 22 (25.4%), no response was obtained in 20 children (23.7%) in 180 day of the therapy. Period of observation was from 12 months to 10.5 years. During this time relapse occurred in 6 patients (7%). We observed 16 deaths: 7 early during the first 3 months of immunosuppressive therapy (IS) and 9 after the first 3 months of IS.


CONCLUSIONS
the actual survival at 10-years, after immunosuppressive therapy is 81.2% in our group. Transformation to leukaemia or myelodysplastic syndrome (MDS) was not observed in any of our patients. We observed one case with paroxysmal nocturnal haemoglobinuria (PNH).
Pawelec
Katarzyna
K
Katedra i Klinika Pediatrii, Hematologii i Onkologii, Akademia Medyczna, ul. Marszalkowska 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-Czepko
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
Kaczmarek-Kanold
Małgorzata
M
Stolarska
Małgorzata
M
Karolczyk
Izabela
I
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*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage; D004305:Dose-Response Relationship, Drug; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000008:administration & dosage; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods*; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D011044:Poland / Q000453:epidemiology; D012189:Retrospective Studies; D012955:Societies, Medical; D016019:Survival Analysis; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D016572:Cyclosporine