Identification of predictive factors for response to intravenous immunoglobulin treatment in children with immune thrombocytopenia

Abstract

Acute immune thrombocytopenia (ITP) is a common benign bleeding disorder of variable etiology characterized by isolated thrombocytopenia. Intravenous immunoglobulin (IVIG) treatment is generally given as an initial treatment to pediatric patients with ITP, but markers predictive of the response to IVIG remain poorly defined. We retrospectively evaluated whether clinical and laboratory findings before treatment could predict response to IVIG and progression to chronic ITP in Japanese children with ITP. Between April 1997 and December 2011, a total of 49 children with newly diagnosed ITP were initially treated with IVIG. Their medical records were retrospectively reviewed. In multivariate analyses, lower white blood cell (WBC) count was the only unfavorable factor for response to IVIG and progression to chronic ITP. Patients with WBC count <7.0 × 10(9)/L had a lower probability of thrombocytopenia-free survival (41 vs. 77 %, P = 0.003) and a higher rate of progression to chronic ITP (29 vs. 6 %, P = 0.040) than those with WBC count ≥7.0 × 10(9)/L. These results suggest that ITP with lower WBC count may represent a distinct subgroup requiring initial treatment other than IVIG.

Predictive factors of response toIVIGin pediatric immune thrombocytopenic purpura

“…In contrast, older age at diagnosis (≥23 months) and lower platelet count (<9.0 × 10 9 /L) were associated with poorer short‐term response, and older age was unfavorable for long‐term outcome, although Morimoto et al . did not identify older age as an unfavorable predictive factor . In the Morimoto et al .…”

“…Recently, Morimoto et al . reported that a lower WBC count at diagnosis, that is, <7.0 × 10⁹/L, was the only unfavorable factor predicting short‐ and long‐term response to IVIG . Therefore, we investigated whether WBC count at diagnosis could be a predictor, but on ROC analysis WBC count <7.0 × 10 9 /L or <8.54 × 10 9 /L (median) was not identified as an unfavorable predictive variable.…”

“…Cumulative proportion of long‐term (1 year) good prognosis was 53.0% (95%CI: 40.8–65.2) on Kaplan–Meier analysis. In addition to gender and age as principal variables, WBC count and platelet count at diagnosis were included as variables, due to the present analysis for short‐term response and a recent published study by Morimoto et al ., which identified these variables as significant . Their association with TFS was analyzed using the Kaplan–Meier method.…”

“…In the present study also, older age, that is, ≥23 months, was identified as an unfavorable predictive variable for both short‐ and long‐term response to IVIG. If a higher dose of IVIG, such as 2 g/kg, like that used by Morimoto et al ., was used for subjects aged ≥23 months from the start of treatment, they might avoid unfavorable short‐ and long‐term outcome. Furthermore, if the IVIG dose was increased, prednisolone may be spared as a first‐line treatment and patient age may not be a risk factor any more.…”

Follow-up of pediatric patients treated by IVIG for Langerhans cell histiocytosis (LCH)-related neurodegenerative CNS disease

“…IVIG possesses multiple immune-modulatory and anti-inflammatory properties, thus it has been employed for various immune-mediated diseases such as immune thrombocytopenia [21] and demyelinating neuropathy [9]. In LCH-related ND-CNS, the exact pathogenesis remains unknown; however, it seems conceivable that it is one of immune-mediated demyelinating disease of CNS as well as paraneoplastic neurological disease.…”