Pediatric Mmune Thrombocytopenic PurpuraITPNCT06601543
Steroid Resistance in Pediatric Immune Thrombocytopenic Purpura
An observational study of Pediatric Mmune Thrombocytopenic Purpura, run by Assiut University.
Trial summary
To Predicting steroid resistance on children newly diagnosed with immune thrombocytopenic purpura Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Pediatric Mmune Thrombocytopenic Purpura
- How many people
- 102 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2024to Nov 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children diagnosed ITP and take steroid an treatment
Who may be able to join
- 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
Who cannot join
- children with immune thrombocytopenic purpura below age of 1 years and above 18 years, patient with thrombocytopenic purpura with secondary causes, and chronic ITP
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Predictors of Steroid Resistance in Pediatric Acute Immune Thrombocytopenic Purpura
To Predicting steroid resistance on children newly diagnosed with immune thrombocytopenic purpura
Read the detailed description
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.
- Conditions
- Pediatric Mmune Thrombocytopenic Purpura
What is being measured
The primary outcome is the main question the study is designed to answer.
predicting steroid resistance on children with acute immune thrombocytopenic purpura in comparison between steroid resistant group and steroid sensitive group Secondary (subsidiary):
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.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 Oct 2024 (estimated)
- Primary completionOct 2025 (estimated)
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Pediatric Mmune Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 102 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shereen Hassan abd Elrady Mohammed, Assiut University
- Investigators
- Azza Ahmed El-Tayab, Principal investigatorMervat Amin Mahmoud, Study director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06601543
- Sponsor's protocol ID
- Steroid resistance in ITP
- Acronym
- ITP
- Status last verified
- Sep 2024
- First submitted
- 15 Sep 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Buchanan GR, Adix L. Grading of hemorrhage in children with idiopathic thrombocytopenic purpura. J Pediatr. 2002 Nov;141(5):683-8. doi: 10.1067/mpd.2002.128547.
PubMed 12410198Su Y, Xu H, Xu Y, Yu J, Dai B, Xian Y, Xiao J. A retrospective analysis of therapeutic responses to two distinct corticosteroids in 259 children with acute primary idiopathic thrombocytopenic purpura. Hematology. 2009 Oct;14(5):286-9. doi: 10.1179/102453309X12473408860343.
PubMed 19843385Celik M, Bulbul A, Aydogan G, Tugcu D, Can E, Uslu S, Dursun M. Comparison of anti-D immunoglobulin, methylprednisolone, or intravenous immunoglobulin therapy in newly diagnosed pediatric immune thrombocytopenic purpura. J Thromb Thrombolysis. 2013 Feb;35(2):228-33. doi: 10.1007/s11239-012-0801-z.
PubMed 22956408Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, Chong BH, Cines DB, Gernsheimer TB, Godeau B, Grainger J, Greer I, Hunt BJ, Imbach PA, Lyons G, McMillan R, Rodeghiero F, Sanz MA, Tarantino M, Watson S, Young J, Kuter DJ. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010 Jan 14;115(2):168-86. doi: 10.1182/blood-2009-06-225565. Epub 2009 Oct 21.
PubMed 19846889Arnold DM. Bleeding complications in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2015;2015:237-42. doi: 10.1182/asheducation-2015.1.237.
PubMed 26637728Kuhne T, Buchanan GR, Zimmerman S, Michaels LA, Kohan R, Berchtold W, Imbach P; Intercontinental Childhood ITP Study Group; Intercontinental Childhood ITP Study Group. A prospective comparative study of 2540 infants and children with newly diagnosed idiopathic thrombocytopenic purpura (ITP) from the Intercontinental Childhood ITP Study Group. J Pediatr. 2003 Nov;143(5):605-8. doi: 10.1067/s0022-3476(03)00535-3.
PubMed 14615730Consolini R, Legitimo A, Caparello MC. The Centenary of Immune Thrombocytopenia - Part 1: Revising Nomenclature and Pathogenesis. Front Pediatr. 2016 Oct 19;4:102. doi: 10.3389/fped.2016.00102. eCollection 2016.
PubMed 27807534Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.
PubMed 31794604Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.
PubMed 19005182Zitek T, Weber L, Pinzon D, Warren N. Assessment and Management of Immune Thrombocytopenia (ITP) in the Emergency Department: Current Perspectives. Open Access Emerg Med. 2022 Jan 29;14:25-34. doi: 10.2147/OAEM.S331675. eCollection 2022.
PubMed 35125895
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictors of Steroid Resistance in Pediatric Acute Immune Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT06601543
- Acronym / sponsor ID
- ITP / Steroid resistance in ITP
- Conditions
- Pediatric Mmune Thrombocytopenic Purpura
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 102 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Oct 2024 (estimated)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Nov 2025 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To Predicting steroid resistance on children newly diagnosed with immune thrombocytopenic purpura
Detailed description
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children diagnosed ITP and take steroid an treatment
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- children with immune thrombocytopenic purpura below age of 1 years and above 18 years, patient with thrombocytopenic purpura with secondary causes, and chronic ITP
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Background: PubMed 12410198Buchanan GR, Adix L. Grading of hemorrhage in children with idiopathic thrombocytopenic purpura. J Pediatr. 2002 Nov;141(5):683-8. doi: 10.1067/mpd.2002.128547.
- Background: PubMed 19843385Su Y, Xu H, Xu Y, Yu J, Dai B, Xian Y, Xiao J. A retrospective analysis of therapeutic responses to two distinct corticosteroids in 259 children with acute primary idiopathic thrombocytopenic purpura. Hematology. 2009 Oct;14(5):286-9. doi: 10.1179/102453309X12473408860343.
- Background: PubMed 22956408Celik M, Bulbul A, Aydogan G, Tugcu D, Can E, Uslu S, Dursun M. Comparison of anti-D immunoglobulin, methylprednisolone, or intravenous immunoglobulin therapy in newly diagnosed pediatric immune thrombocytopenic purpura. J Thromb Thrombolysis. 2013 Feb;35(2):228-33. doi: 10.1007/s11239-012-0801-z.
- Background: PubMed 19846889Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, Chong BH, Cines DB, Gernsheimer TB, Godeau B, Grainger J, Greer I, Hunt BJ, Imbach PA, Lyons G, McMillan R, Rodeghiero F, Sanz MA, Tarantino M, Watson S, Young J, Kuter DJ. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010 Jan 14;115(2):168-86. doi: 10.1182/blood-2009-06-225565. Epub 2009 Oct 21.
- Background: PubMed 26637728Arnold DM. Bleeding complications in immune thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2015;2015:237-42. doi: 10.1182/asheducation-2015.1.237.
- Background: PubMed 14615730Kuhne T, Buchanan GR, Zimmerman S, Michaels LA, Kohan R, Berchtold W, Imbach P; Intercontinental Childhood ITP Study Group; Intercontinental Childhood ITP Study Group. A prospective comparative study of 2540 infants and children with newly diagnosed idiopathic thrombocytopenic purpura (ITP) from the Intercontinental Childhood ITP Study Group. J Pediatr. 2003 Nov;143(5):605-8. doi: 10.1067/s0022-3476(03)00535-3.
- Background: PubMed 27807534Consolini R, Legitimo A, Caparello MC. The Centenary of Immune Thrombocytopenia - Part 1: Revising Nomenclature and Pathogenesis. Front Pediatr. 2016 Oct 19;4:102. doi: 10.3389/fped.2016.00102. eCollection 2016.
- Background: PubMed 31794604Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.
- Background: PubMed 19005182Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.
- Background: PubMed 35125895Zitek T, Weber L, Pinzon D, Warren N. Assessment and Management of Immune Thrombocytopenia (ITP) in the Emergency Department: Current Perspectives. Open Access Emerg Med. 2022 Jan 29;14:25-34. doi: 10.2147/OAEM.S331675. eCollection 2022.
- Related InfoLink provided by the sponsor.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Azza Ahmed El-Tayab principal investigator
Mervat Amin Mahmoud study director
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Mmune Thrombocytopenic Purpura, run by Assiut University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Shereen Hassan Abd -Elrady (00201024567924 ext. 00201018742203, shrinhsn122@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06601543, which also lists 10 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.