title: [Results of immunosuppressive therapy in children with severe aplastic anaemia. Report by the Polish Paediatric Leukaemia and Lymphoma Study Group].
abstract: 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).
authors.affiliation: Katedra i Klinika Pediatrii, Hematologii i Onkologii, Akademia Medyczna, ul. Marszalkowska 24, 00-576 Warszawa, Poland. katpawelec@poczta.onet.pl
authors.forename: Katarzyna
authors.initials: K
authors.lastname: Pawelec
authors.forename: Michał
authors.initials: M
authors.lastname: Matysiak
authors.forename: Edyta
authors.initials: E
authors.lastname: Niewiadomska
authors.forename: Roma
authors.initials: R
authors.lastname: Rokicka-Milewska
authors.forename: Jerzy
authors.initials: J
authors.lastname: Kowalczyk
authors.forename: Jolanta
authors.initials: J
authors.lastname: Stefaniak
authors.forename: Walentyna
authors.initials: W
authors.lastname: Balwierz
authors.forename: Ewa
authors.initials: E
authors.lastname: Załecka-Czepko
authors.forename: Alicja
authors.initials: A
authors.lastname: Chybicka
authors.forename: Krzysztof
authors.initials: K
authors.lastname: Szmyd
authors.forename: Danuta
authors.initials: D
authors.lastname: Sońta-Jakimczyk
authors.forename: Halina
authors.initials: H
authors.lastname: Bubała
authors.forename: Agnieszka
authors.initials: A
authors.lastname: Krauze
authors.forename: Mariusz
authors.initials: M
authors.lastname: Wysocki
authors.forename: Andrzej
authors.initials: A
authors.lastname: Kurylak
authors.forename: Jacek
authors.initials: J
authors.lastname: Wachowiak
authors.forename: Małgorzata
authors.initials: M
authors.lastname: Kaczmarek-Kanold
authors.forename: Małgorzata
authors.initials: M
authors.lastname: Stolarska
authors.forename: Izabela
authors.initials: I
authors.lastname: Karolczyk
authors.forename: Maryna
authors.initials: M
authors.lastname: Krawczuk-Rybak
authors.forename: Elzbieta
authors.initials: E
authors.lastname: Leszczyńska
authors.forename: Tomasz
authors.initials: T
authors.lastname: Urasiński
authors.forename: Jarosław
authors.initials: J
authors.lastname: Peregud-Pogorzelski
authors.forename: Anna
authors.initials: A
authors.lastname: Balcerska
authors.forename: Marek
authors.initials: M
authors.lastname: Włazłowski
mesh_terms: 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
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D016572:Cyclosporine