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NCT Number: NCT06601543

Steroid Resistance in Pediatric Immune Thrombocytopenic Purpura

To Predicting steroid resistance on children newly diagnosed with immune thrombocytopenic purpura

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Key information

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Observational

About this study

Immune thrombocytopenia (ITP, platelet counts < 100 × 109/L) is the most common acquired childhood bleeding disorder, clinically characterized by a low platelet count in the absence of other thrombocytopenia causes [1,2].

The estimated incidence of ITP is 100 cases out of a million people per year; about half of these cases occur in previously healthy children, where it represents the most frequent blood disorder [3].

Most children present with a typical history of acute purpura and bruising after a mild viral infection [4]. In severe cases, intracranial hemorrhage (the most 0.5% serious complication, but also the rarest occurring in adults), gastrointestinal hemorrhage in 1.5 % of children, and genitourinary hemorrhage may occur [5].

The International Working Group on ITP defines ITP according to the following clinical phases [6]. These are as follows:

Newly diagnosed ITP is in the first three months post-diagnosis. Persistent ITP is for 3-12 months. Chronic ITP is for > 12 months. Refractory ITP is the failure to restore count of platelet after splenectomy. For children requiring therapy but without life threatening bleeding, corticosteroids are the recommended first line therapy over IVIG or anti-D [2].

Guidelines from the American Society of Hematology recommend a 5-7-day course of prednisone dosed at 2-4 mg/kg/day [2]. Seventy-five percent of children respond to steroids, with platelets recovering to hemostatic range by 2-7 days [7]. If a more rapid rise in platelets is desired, IV methylprednisolone may be used. Studies comparing outcomes between anti-D versus methylprednisolone [8] and comparing methylprednisolone with dexamethasone [9] showed similar response rates with minor side effects in all groups.

A study shows that 98% of patients with corticosteroid exposure experienced one or more side events, and 38% of patients need to stop or reduce corticosteroid therapy [10].

This research aims to develop a new prediction model to evaluate whether newly ITP patients are at high-risk of corticosteroid resistance, and help clinicians to choose better therapy so we divide patients to two groups, steroid response and steroid resistance.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All patients diagnosed as acute immune thrombocytopenic purpura based on clinical manifestations and laboratory investigations from age of 1 years to age of 18 years.

Gender: both six

Exclusion criteria

  • children with immune thrombocytopenic purpura below age of 1 years and above 18 years, patient with thrombocytopenic purpura with secondary causes, and chronic ITP

Treatment and study plan

Primary outcomes

  1. predicting steroid resistance on children with acute immune thrombocytopenic purpura in comparison between steroid resistant group and steroid sensitive group Secondary (subsidiary):

    Time frame: Baseline

    predicting steroid resistance on children with acute immune thrombocytopenic purpura in comparison between steroid resistant group and steroid sensitive group to allow early introduction of alternative therapy before bleeding symptoms occurs and to avoid side effects of steroid use.

Study contacts

Contact information is provided by the study sponsor or research team.

Shereen Hassan Abd -Elrady

CONTACT

[email protected]

00201024567924 ext. 00201018742203

Sponsors and collaborators

Lead sponsor

Assiut University

Other

Registry information

Official study title

Predictors of Steroid Resistance in Pediatric Acute Immune Thrombocytopenic Purpura

Acronym: ITP

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Sep 19, 2024
Registry last updated
Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.