Management of chronic immune thrombocytopenia in children and adolescents: lessons from an Austrian national cross-sectional study of 81 patients.
Chronic immune thrombocytopenia (cITP) is often associated with an underlying predisposition towards autoimmunity, recognition of which is relevant to guide treatment. International recommendations on diagnostic steps and therapeutic measures of cITP in childhood exist. However, due to the low prevalence (1-2/100,000) and a variation of availability of immunological and hematological tests and treatments across pediatric units, we postulated that these guidelines are not uniformly adhered to and that immune dysregulation syndromes remained undiscovered. To delineate the current management of children and adolescents with cITP in Austria, we performed a nationwide cross-sectional study. Between 2011 and 2014, 81 children with cITP were seen at seven centers (median age 8.75 years; range 1-17; female:male ratio 47:34) at 641 visits during 180 patient years after diagnosis of cITP (>12 months ITP duration). Additional diagnoses were noted, most frequently immune or autoimmune disorders, hematologic diseases, or infections (in 37.3%, including Evans syndrome, autoimmune lymphoproliferative syndrome, systemic lupus erythematosus, and Fanconi anemia), or other symptoms like bi- or pancytopenia (n=9), lymphoproliferation or granulomatous inflammation (n = 3). Both decision to treat as well as choice of treatment varied: smaller centers tended to observe more frequently, larger centers applied a pattern of treatment modalities that appeared to depend less on bleeding tendency than on center policy. More than 50% of therapeutic interventions occurred in bleedings scores ≤2 (of 5), suggesting a strong psychosocial intention to treat. Platelet increment upon 479 therapeutic interventions of eight types was evaluated, with multiple treatment approaches being pursued sequentially in refractory patients. These data confirm the hypothesis of heterogeneous diagnostic and therapeutic management of cITP in Austrian children and corroborate the need for (1) a precise panel of parameters to exclude underlying disorders and (2) for biomarkers to predict treatment response.
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria; Research Unit for Pediatric Hematology and Immunology, Medical University Graz, Austria.
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Sipurzynski
Division of Pediatric Hematology-Oncology at St. Anna Children's Hospital, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Austria.
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B
Fahrner
Department of Pediatrics and Adolescent Medicine, Landesklinikum Hochsteiermark, Leoben, Austria.
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R
Kerbl
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Innsbruck, Austria.
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Crazzolara
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Landeskrankenhaus Salzburg, Austria.
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Jones
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Landeskrankenhaus Linz, Austria.
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Ebetsberger
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Landeskrankenhaus Klagenfurt, Austria.
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B
Jauk
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria.
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V
Strenger
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria; Research Unit for Pediatric Hematology and Immunology, Medical University Graz, Austria.
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B
Wohlmuther
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria.
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W
Schwinger
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria.
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Lackner
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria.
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Urban
Division of Pediatric Hematology-Oncology at St. Anna Children's Hospital, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Austria.
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Holter
Division of Pediatric Hematology-Oncology at St. Anna Children's Hospital, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Austria; Department of Pediatrics and Adolescent Medicine, Krankenhaus Rudolfstiftung, Vienna, Austria.
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M
Minkov
Division of Pediatric Hematology-Oncology at St. Anna Children's Hospital, Department of Pediatrics and Adolescent Medicine, Medical University Vienna, Austria.
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L
Kager
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria. Electronic address: martin.benesch@klinikum-graz.at.
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M
Benesch
Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria; Research Unit for Pediatric Hematology and Immunology, Medical University Graz, Austria.
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Seidel
D000293:Adolescent; D001317:Austria; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D003430:Cross-Sectional Studies; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*
Autoimmune cytopenia; Autoimmune lymphoproliferative syndrome; Consensus guidelines; Evans syndrome; Immune thrombocytopenia (ITP); Systemic lupus erythematosus