Medycyna Wieku Rozwojowego

832
Katarzyna Pawelec i wsp.
Wnioski: prawdopodobieƒstwo 10-letniego prze˝ycia po leczeniu immunosupresyjnym
w naszej grupie wynosi 81,2%. U ˝adnego dziecka nie nastàpi∏a transformacja w ostrà bia-
∏aczk´ ani zespó∏ mielodysplastyczny (MDS). U jednego dziecka stwierdziliÊmy nocnà napadowà
hemoglobinuri´ (PNH).
S∏owa kluczowe: anemia aplastyczna, leczenie immunosupresyjne, dzieci
RESULTS OF IMMUNOSUPRESSIVE THERAPY
IN CHILDREN WITH SEVERE APLASTIC ANAEMIA.
REPORT BY THE POLISH PAEDIATRIC LEUKAEMIA
AND LYMPHOMA STUDY GROUP
Abstract. Introduction: 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.
Aim: evaluation of results of immunosupressive therapy in children with severe aplastic
anaemia.
Material and 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, prednisolon
and granulocyto- or granulocytomacrophagic-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 immunosupressive
therapy (IS) and 9 after the first 3 months of IS.
Conclusion: the actual survival at 10-years, after immunosupressive 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).
Key words: aplastic anaemia, immunosppressive therapy, children
WST¢P
Nabyta anemia aplastyczna (aplastic anemia-AA) jest rzadkà chorobà wieku
dzieci´cego charakteryzujàcà si´ pancytopenià krwi obwodowej oraz hipoplazjà szpiku
kostnego (1-5). Wcià˝ w oko∏o 50% przypadków nie jest mo˝liwe ustalenie przyczyny
choroby. Mechanizm immunologiczny jest podstawà patogenezy AA. W wyniku
dzia∏ania zaktywowanych cytotoksycznych limfocytów T (CD8), produkujàcych interferon
gamma (IFN-γ) i czynnik martwicy nowotworów (TNF) oraz interleukin´-2 (IL-2),
dochodzi do apoptozy komórek macierzystych (1-8). Terapia immunosupresyjna (IS)
odnosi bardzo dobre rezultaty poprzez blokowanie mechanizmów immunologicznych