A comparative prospective observational study of children and adults with immune thrombocytopenia: 2‐year follow‐up

Abstract

Comparative clinical studies of children and adults with immune thrombocytopenia (ITP) are poorly covered in the literature. However, the accepted classification of ITP-childhood ITP and adult ITP-results in considerable differences in treatment protocols and practice guidelines. The analysis of the Pediatric and Adult Registry on Chronic ITP (PARC-ITP) of patients at first presentation demonstrated fewer differences in clinical and laboratory findings at initial diagnosis between children and adults than expected. The present report of 2-year follow-up data supports the hypothesis that there are common aspects of childhood and adult ITP. Data of 3360 children and 420 adults were collected during the time of 2004 until 2015 at initial diagnosis. Follow-up information was available for 51% and 33% of children and 66% and 49% of adults at 12- and 24-months, respectively. Similarities were found in unexpected areas of ITP, such as the rate of late remission at 12 and 24 months, reported bleeding sites, platelet count in bleeders, and the frequency of treated patients with persistent or chronic ITP. Differences were confirmed for the overall rate of remission and treatment modalities. Unexpected differences were found in the percentage of nonbleeders, with more adults in the nonbleeder group. More studies are needed to investigate different age groups with the aim to optimize their management.

Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia

“…2 However, these children often have very low platelet counts that are very difficult to treat, have an ongoing risk of intracranial hemorrhage and other bleeding, and have an impaired quality of life. 3,4 There are few data on long-term improvement beyond two years of disease, 5,6 and all major centers are familiar with patients with very long-term (i.e. of many years’ duration) refractory chronic ITP for whom they have no good treatment options.…”

Second‐line treatments in children with immune thrombocytopenia: Effect on platelet count and patient‐centered outcomes

“…Current guidelines for the management of newly diagnosed patients with ITP suggest observation in the absence of bleeding symptoms, or treatment with standard first‐line therapies (corticosteroids, anti‐RhD immunoglobulin, or intravenous immunoglobulin [IVIG]) for those with bleeding symptoms . In the Pediatric and Adult Registry on Chronic ITP, 38‐47% of the children who received upfront treatment required second‐line therapies at 6‐24 months . Reasons for treating patients with second‐line therapies may include refractory disease, bleeding symptoms or risk of bleeding, poor perceived quality of life, need for peri‐procedural management, and/or desire to achieve remission.…”

Trombocitopenia inmune. Guía de diagnóstico y tratamiento

“…Sin embargo, hay varios factores que tienen distinto valor predictivo. [18][19][20][21][22][23][24][25][26][27][28][29][30][31][32][33] El principal es la edad. Los niños menores de 12 meses constituyen un grupo pronóstico especial, caracterizado por la corta duración de la enfermedad y un muy elevado porcentaje de remisiones (aproximadamente, el 90 %), que no es influenciado por otros factores pronósticos.…”

“…La muerte, debida a un sangrado grave en los órganos vitales -casi excluyentemente, hemorragia intracraneana-, se observa en alrededor del 0,2 % de los pacientes. 19,21,[23][24][25]32,34,35…”