What side effects are associated with this type of intervention?

Common treatments for Thrombocytopenic Purpura (ITP) include corticosteroids, intravenous immunoglobulin (IVIG), and thrombopoietin receptor agonists such as eltrombopag and romiplostim. Corticosteroids can cause weight gain, hypertension, hyperglycemia, and increased risk of infections. IVIG may lead to headaches, fever, chills, and allergic reactions. Eltrombopag and romiplostim, which enhance platelet production, can cause side effects like liver function abnormalities, thromboembolic events, and headaches. Patients should discuss these potential side effects with their healthcare provider to make informed treatment decisions.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Thrombocytopenic Purpura (ITP) that focus on immune modulation and platelet production enhancement. Eltrombopag and romiplostim are thrombopoietin receptor agonists that stimulate platelet production and have been approved for use in ITP. Eltrombopag is also approved for use in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Romiplostim is approved for chronic ITP in patients who have had an insufficient response to other treatments. Both drugs have shown efficacy in increasing platelet counts and reducing bleeding events in clinical trials.

What other types of research exist?

Promising novel alternatives for treating ITP in 2024 include thrombopoietin receptor agonists such as eltrombopag and romiplostim, which enhance platelet production. Additionally, avatrombopag and lusutrombopag are newer agents that potentiate the effect of endogenous thrombopoietin and may be beneficial in myeloablative settings. These treatments have shown efficacy in increasing platelet counts and reducing the need for platelet transfusions.

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Thrombopoietin Receptor Agonist

Eltrombopag vs Standard Treatment for ITP in Children

Phase 3

Waitlist Available

Led By Jenny Despotovic, DO

Research Sponsored by Baylor College of Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Upfront treatment: Patient within 10 days of ITP diagnosis who has not received previous treatment OR Treatment failure: Patients who have failed standard management (observation or treatment with one or more first-line agents)

Treatment options include one of three standard therapies, (IVIg, steroids, or Anti-D). For example, if patient has previously shown no response to IVIg or steroids and is Rh-negative, patient would not be eligible for study.

Must not have

Impaired cardiac function

Severe bleeding: Buchanan Overall Grade 4 or 5 bleeding, or severe bleeding requiring emergent treatment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

All Individual Drugs Already Approved

Pivotal Trial

No Placebo-Only Group

Summary

This trial tests eltrombopag, a drug that helps make more platelets, in children with a specific blood condition. The goal is to see if it works better than standard treatments.

Who is the study for?

This trial is for children aged 1 to less than 18 with newly diagnosed ITP, a condition causing low platelet count. They must not have received prior treatment or have failed standard treatments like IVIG, steroids, or Anti-D. Participants need pharmacologic treatment and should be able to return for lab studies.

What is being tested?

The study compares Eltrombopag against three standard therapies (IVIG, Rho(D) Immune Globulin, Steroids) in treating ITP in children. It's an open label phase 3 trial where participants are randomly assigned to receive either the new intervention or one of the standard options.

What are the potential side effects?

Eltrombopag may cause liver issues, infections that don't respond well to drugs, heart problems if there's pre-existing cardiac disease, bleeding risks especially if severe bleeding has occurred before and potential cataract development.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I was diagnosed with ITP within the last 10 days and haven't been treated, or my initial ITP treatments didn't work.

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I have not responded to IVIg, steroids, or Anti-D and am Rh-negative.

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I am younger than 18 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart does not function properly.

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I have experienced severe bleeding that needed emergency care.

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I have Evans Syndrome with ongoing breakdown of red blood cells.

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I have a heart condition that could make participating in the study unsafe.

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I have an infection that isn't getting better with treatment.

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I have risk factors for blood clots.

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I have been diagnosed with HIV or Hepatitis C.

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I have kidney problems.

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I have previously been treated with TPO-RA (eltrombopag or romiplostim).

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I have been diagnosed with cancer.

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I have ITP caused by another condition like lupus.

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I have had a stem cell or solid organ transplant.

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I am taking medication to prevent blood clots.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of patients with a platelet response

Secondary study objectives

Bleeding Score

Blood Iron values

Hockenberry Fatigue Scale-Parent

+7 more

Other study objectives

Change in Hockenberry fatigue

Extreme thrombocytosis

Global Change Scale scores

+7 more

Side effects data

From 2014 Phase 3 trial • 92 Patients • NCT01520909

17%

Nasopharyngitis

16%

Rhinitis

13%

Epistaxis

11%

Upper respiratory tract infection

11%

Cough

10%

Headache

10%

Abdominal pain

Pyrexia

Aspartate Aminotransferase increased

Alanine Aminotransferase increased

Decreased appetite

Vitamin D deficiency

Abdominal pain upper

Oropharyngeal pain

Rash

Toothache

Diarrhoea

Activated partial thromboplastin time prolonged

Blood alkaline Phosphatase increased

Blood creatinine increased

Bronchitis

Contusion

Gingival bleeding

Mouth haemorrhage

Nausea

Rhinorrhoea

Vomiting

Pneumonia fungal

Impetigo

Furuncle

Dyspepsia

Retinal vascular disorder

Constipation

Excoriation

Paraesthesia

Soft tissue injury

Cellulitis

Anaemia

Allergy to chemicals

Ear pain

Rash pruritic

Dermatitis allergic

Gingivitis

Groin pain

Osteoporosis

Influenza like illness

Lip haemorrhage

Menorrhagia

Viral pharyngitis

Pneumonia

Influenza

Joint injury

Lice infestation

Motion sickness

Pharyngitis

Platelet count increased

Somnolence

Subcutaneous abscess

Tongue haemorrhage

Tonsillar hypertrophy

Meningitis aseptic

Alanine aminotransferase abnormal

Aspartate aminotransferase abnormal

Gastritis

Asthenia

Back pain

Bronchospasm

Bulimia nervosa

Non-cardiac chest pain

Pain

100%

80%

60%

40%

20%

Study treatment Arm

Part 1: Placebo

Part 1: Eltrombopag

Part 2: Eltrombopag

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: EltrombopagExperimental Treatment1 Intervention

Patients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response.

Group II: Standard first-line therapyActive Control3 Interventions

Subjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm. Standard therapy will be administered as commercially available drug. Investigator may choose amongst the following: * IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) * Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days * Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Eltrombopag

FDA approved

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Thrombocytopenic Purpura (ITP) include immune modulation and platelet production enhancement. Corticosteroids, such as prednisone, work by suppressing the immune system to reduce the destruction of platelets. Intravenous immunoglobulin (IVIG) provides a temporary increase in platelet count by interfering with the immune system's ability to destroy platelets. Thrombopoietin receptor agonists like eltrombopag and romiplostim stimulate the bone marrow to produce more platelets. These treatments are crucial for ITP patients as they help manage bleeding risks and improve platelet counts, thereby enhancing the patient's quality of life and reducing the likelihood of severe bleeding complications.