Thrombocytopenia and aplastic anemia in Finnish children and adolescents
Thesis event information
Date and time of the thesis defence
Topic of the dissertation
Thrombocytopenia and aplastic anemia in Finnish children and adolescents
Doctoral candidate
Medical Doctor Lauri-Matti Kulmala
Faculty and unit
University of Oulu Graduate School, Faculty of Medicine, Research Unit of Clinical Medicine
Subject of study
Medicine
Opponent
Docent Samppa Ryhänen, University of Helsinki
Custos
Docent Riitta Niinimäki, University of Oulu
Thrombocytopenia and aplastic anemia in Finnish children and adolescents
Childhood thrombocytopenia is often caused by immune thrombocytopenia (ITP) but may also be a sign of an underlying disorder. Aplastic anemia (AA) in children is a rare, life-threatening condition characterized by pancytopenia due to bone marrow failure resulting from immune mechanisms. Similar phenotypes of pancytopenia may also result from refractory cytopenia of childhood (RCC) or inherited bone marrow failure syndromes (IBMFS). Childhood AA has incompletely known effects on health-related quality of life (HRQoL).
The aims of this work were to examine differences in presentation between ITP and inherited platelet disorders or primary immunodeficiencies; to describe diagnostic and treatment strategies in childhood ITP and identify factors associated with chronic disease; to determine the incidence of childhood acquired AA (AAA) in Finland and characterize the phenotype, treatment and survival of children with AAA and other disorders fulfilling blood count criteria for AA; and to assess HRQoL in childhood AA survivors.
Finnish children presenting with isolated thrombocytopenia or AA between 2006 and 2020 were included in population-based cohort studies. Patient files from pediatric hemato-oncology units were reviewed and patients reclassified using current diagnostic criteria and definitions. HRQoL was assessed in AA survivors using generic and disease-specific questionnaires.
Among the 506 children with isolated thrombocytopenia, ITP was the final diagnosis in 80%. A platelet count below 12 × 10⁹/L was highly suggestive for ITP. Severe bleeding was rare. Most patients recovered under observation, while 26% developed chronic disease. The incidence of AAA was 2.9/million children/year. Of the 52 patients fulfilling blood count criteria for AA, 41 had AAA, 5 IBMFS and 6 RCC. Most patients responded to treatment and overall survival was 90%. HRQoL in childhood AA was generally good, but fear of disease progression and burden of follow-up persisted, particularly among female patients.
In conclusion, thrombocytopenia in children is usually a transient condition with a good prognosis; patients with higher platelet counts and an insidious onset are more likely to have other conditions than ITP. AA is a rare disorder, and the overall survival is good. HRQoL of children with AA is good, but emotional distress is often observed.
The aims of this work were to examine differences in presentation between ITP and inherited platelet disorders or primary immunodeficiencies; to describe diagnostic and treatment strategies in childhood ITP and identify factors associated with chronic disease; to determine the incidence of childhood acquired AA (AAA) in Finland and characterize the phenotype, treatment and survival of children with AAA and other disorders fulfilling blood count criteria for AA; and to assess HRQoL in childhood AA survivors.
Finnish children presenting with isolated thrombocytopenia or AA between 2006 and 2020 were included in population-based cohort studies. Patient files from pediatric hemato-oncology units were reviewed and patients reclassified using current diagnostic criteria and definitions. HRQoL was assessed in AA survivors using generic and disease-specific questionnaires.
Among the 506 children with isolated thrombocytopenia, ITP was the final diagnosis in 80%. A platelet count below 12 × 10⁹/L was highly suggestive for ITP. Severe bleeding was rare. Most patients recovered under observation, while 26% developed chronic disease. The incidence of AAA was 2.9/million children/year. Of the 52 patients fulfilling blood count criteria for AA, 41 had AAA, 5 IBMFS and 6 RCC. Most patients responded to treatment and overall survival was 90%. HRQoL in childhood AA was generally good, but fear of disease progression and burden of follow-up persisted, particularly among female patients.
In conclusion, thrombocytopenia in children is usually a transient condition with a good prognosis; patients with higher platelet counts and an insidious onset are more likely to have other conditions than ITP. AA is a rare disorder, and the overall survival is good. HRQoL of children with AA is good, but emotional distress is often observed.
Created 16.3.2026 | Updated 16.3.2026