Predictive factors of response to<scp>IVIG</scp>in pediatric immune thrombocytopenic purpura

Higashide, Yukiko; Hori, Tsukasa; Yoto, Yuko; Kabutoya, Hiroyuki; S, Honjo; Sakai, Yoshiyuki; Nojima, Masanori; Yoda, Minami; Yamamoto, Masaki; Tsutsumi, Hiroyuki

doi:10.1111/ped.13505

Cited by 9 publications

(9 citation statements)

References 13 publications

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“…A recent Japanese study showed that children ,23 months of age (n 5 24) were more likely to achieve a better PC response at the 2-week mark following therapy than older children ($23 months; n 5 29). 35 However, in that study, the initial rise in PC in the first 24 hours was not described. We found a trend for older children to show a less brisk increment in PC.…”

Section: Discussionmentioning

confidence: 77%

IVMP+IVIG raises platelet counts faster than IVIG alone: results of a randomized, blinded trial in childhood ITP

et al. 2020

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Children with immune thrombocytopenia (ITP) rarely suffer from life-threatening bleeds (eg, intracranial hemorrhage). In such settings, the combination of IV methylprednisolone (IVMP) with IV immune globulin (IVIG) is used to rapidly increase platelet counts (PCs). However, there are no controlled data to support using combination therapy over IVIG alone. We conducted a randomized, double-blind, placebo-controlled study to evaluate the rapidity of the PC increment and associated adverse events (AEs) between 2 regimens: A (IV placebo) and B (IVMP 30 mg/kg), both given over 1 hour, followed in both cases by IVIG (Gamunex 10%) 1 g/kg over 2-3 hours in children 1-17 years old with primary ITP and PCs <20 × 109/L in whom physicians had decided to treat with IVIG. Thirty-two children (ages: median, 8 years; range, 1.2-17.5 years) with a mean baseline PC of 9.2 × 109/L participated. Eighteen were randomized to regimen A and 14 to regimen B. By 8 hours after initiating therapy, 55% of all children had a PC ≥20 × 109/L (no group difference). By 24 hours, mean PCs were 76.9 × 109/L (B) vs 55 × 109/L (A) (P = .06; P = .035 when adjusted for intergroup differences in patient ages). No patient experienced severe bleeding/unexpected severe AEs. There were statistically fewer IVIG-related headaches in the group receiving combination therapy (P = .046). Our findings show a rapid response to IVIG with/without steroids and provide evidence to support the use of IVMP+IVIG in life-threatening situations. This trial was registered at www.clinicaltrials.gov as #NCT00376077.

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Section: Discussionmentioning

confidence: 77%

IVMP+IVIG raises platelet counts faster than IVIG alone: results of a randomized, blinded trial in childhood ITP

et al. 2020

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“…Notably, demographic (age, sex), clinical (disease onset, preceding infection or vaccination), or biological (platelet counts at diagnosis) were already reported to be not only associated with (11), but also predictive of prolonged disease (13). Besides, previous publications used linear regression to describe platelet count evolution over time but reported conflicting results, this evolution being related (14) or not (15) with disease evolution.…”

Section: Discussionmentioning

confidence: 99%

“…Choi et al found that patients with chronic ITP had significantly lower platelet counts one and three months after diagnosis than patients with acute ITP (14). On the contrary, Higashide et al found no association between ITP progression and platelet counts at 12 months (15).…”

Section: Introductionmentioning

confidence: 99%

Prediction of the clinical course of immune thrombocytopenia in children by platelet kinetics

Lejeune

Raoult

Dubrasquet

et al. 2022

Preprint

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Introduction: Childhood immune thrombocytopenia (ITP) is a rare autoimmune disorder characterized by isolated thrombocytopenia. Prolonged ITP (persistent and chronic) leads to a reduced quality of life for children in many domains. To provide optimal support for children, with ITP, it is important to be able to predict those who will develop prolonged ITP. This study aimed to develop a mathematical model based on platelet recovery that allows the early prediction of prolonged ITP. Methods: In this retrospective study, we used platelet counts from the six months following the diagnosis of ITP to model the kinetics of platelet evolution using a pharmacokinetic-pharmacodynamic model. Results: In a learning set (n=103), platelet counts were satisfactorily described by our kinetic model. The K parameter, which describes spontaneous platelet recovery, allowed a distinction between acute and prolonged ITP with an AUC of 0.74. In a validation set (n=58), spontaneous platelet recovery was robustly predicted using platelet counts from 15 (AUC=0.76) or 30 (AUC=0.82) days after ITP diagnosis. Discussion: In our model, platelet recovery quantified using the k parameter allowed prediction of the clinical course of ITP. Future prospective studies are needed to improve the predictivity of this model, in particular, by combining it with the predictive scores previously reported in the literature.

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“…10,37 However, these variants are rare and insufficient to explain the large proportion of non-responders. Retrospective case series have suggested that IVIg-resistant patients have higher rates of leukocyte count ≤7 × 10 9 /L, 47 age above 2 years, a platelet count <9 × 10 9 /L, 48 and a higher expression of IFNγ in peripheral blood mononuclear cells. 49 Although the absence of leukocytosis could be associated with a noninflammatory phenotype, potentially pointing to the absence of autoinflammatory or postinfectious reactive autoimmunity, this is in contrast with the increased IFNγ expression.…”

Section: Findings Of Othersmentioning

confidence: 99%

Biological stratification of clinical disease courses in childhood immune thrombocytopenia

Schmidt

Heitink‐Pollé

Mertens

et al. 2021

Journal of Thrombosis and Haemostasis

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Predictive factors of response toIVIGin pediatric immune thrombocytopenic purpura

Abstract: For new-onset ITP in patients aged >2 years, corticosteroid therapy in addition to IVIG may be considered as the initial treatment.

Cited by 9 publications

References 13 publications

IVMP+IVIG raises platelet counts faster than IVIG alone: results of a randomized, blinded trial in childhood ITP

IVMP+IVIG raises platelet counts faster than IVIG alone: results of a randomized, blinded trial in childhood ITP

Prediction of the clinical course of immune thrombocytopenia in children by platelet kinetics

Biological stratification of clinical disease courses in childhood immune thrombocytopenia

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