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Areas of Research ITP and Immune Hematology

<p>ITP and Immune Hematology</p>

Our Research Focus

Our team is actively investigating the causes, progression, and treatment of immune thrombocytopenia (ITP) and related immune-mediated blood disorders. Through collaborations with leading centers across the United States, we are advancing research that improves outcomes and expands treatment options for children with these conditions. 

Transforming Care Through Clinical Trials

The PINES Clinical Trial 
Investigators at Texas Children's led the PINES trial (Pediatric ITP Newly diagnosed patients treated with Eltrombopag vs Standard therapy), a phase 3 randomized clinical trial conducted across 23 centers in the Pediatric ITP Consortium of North America (ICON). This study evaluated eltrombopag as a frontline treatment for children newly diagnosed with ITP who required medication but did not have severe bleeding. Eltrombopag, a thrombopoietin receptor agonist already FDA-approved for chronic ITP, was compared with standard therapy in 118 children with newly diagnosed ITP. 

The results were striking: among 118 enrolled patients, 65% of those receiving eltrombopag achieved a sustained platelet response, compared with 35% receiving standard therapy.  Because the prespecified efficacy threshold was reached early, the trial was stopped at interim analysis and the findings were published in JAMA.

What This Means for Patients: Eltrombopag offers a meaningful, sustained response option for children with newly diagnosed ITP who require treatment for mild to moderate bleeding or quality‑of‑life concerns.

Advancing Discovery Through the ICON BioBank

A National Resource Housed at Texas Children’s 

Two of the most common questions families ask after an ITP diagnosis are: "What caused me to develop ITP?" and "Will my ITP go away?" 

To help answer these questions, Texas Children’s investigators partnered will colleagues across North America to establish the ITP Consortium of North America (ICON) BioBank in 2015.

Now the largest pediatric ITP biorepository in North America, the ICON Biobank includes nearly 1,000 patients and is operated through a partnership of Texas Children's Hospital and eight children's hospitals across the United States.

The biobank collects DNA, RNA, and plasma samples at multiple points throughout a patient's disease course, creating a unique resource for understanding disease mechanisms, identifying risk factors, and developing more targeted treatments.     

Research using samples from the ICON BioBank has led to important discoveries:

Our Impact: The ICON BioBank is driving discoveries that link genetic risk, immune dysregulation, and clinical outcomes in ITP.

Collaborations and Community Engagement

Immune hematology researchers are conducting lab-based research to investigate variability in treatment response to commonly used front-line agents in ITP. Additionally, we are working on several projects to improve our understanding of the natural history of autoimmune hemolytic anemia and ITP in children, specifically the association with other autoimmune disorders such as systemic lupus erythematosus (SLE) and common variable immune deficiency (CVID). 

Our Program is a member of the ITP Consortium of North America (ICON), a national research network of pediatric hematologists dedicated to advancing the understanding and treatment of ITP through multicenter collaboration. We also partner with the Platelet Disorder Support Association (PDSA), contributing educational content to The Platelet News on topics including results from the PINES trial, the genetics of ITP, and the evolving terminology for refractory ITP in children.

Why This Matters: By combining innovative research, national collaboration, and patient-focused education, we help translate scientific discoveries into better care for children with immune-mediated blood disorders.  

Our Researchers

Areas of Research in Cancer and Blood Disorders

Adolescent Hematology and Gynecology Bone Marrow Failure Bone Tumors Brain and Spinal Cord Tumors Cancer Genetics and Genomics Cancer Survivorship Cell and Gene Therapy Cell Therapy and Bone Marrow Transplant Developmental Therapeutics Epidemiology (Cancer and Hematology) Hemostasis and Thrombosis Histiocytosis ITP and Immune Hematology Immunotherapy Leukemia Liver Tumors Lymphoma Neuroblastoma Precision Oncology Rare Tumors Retinoblastoma Sickle Cell Disease and Thalassemia Soft Tissue Sarcoma Solid Tumors Thyroid Tumors
Adolescent Hematology and Gynecology Bone Tumors Cancer Genetics and Genomics Cell and Gene Therapy Developmental Therapeutics Hemostasis and Thrombosis ITP and Immune Hematology Leukemia Lymphoma Precision Oncology Retinoblastoma Soft Tissue Sarcoma Thyroid Tumors Bone Marrow Failure Brain and Spinal Cord Tumors Cancer Survivorship Cell Therapy and Bone Marrow Transplant Epidemiology (Cancer and Hematology) Histiocytosis Immunotherapy Liver Tumors Neuroblastoma Rare Tumors Sickle Cell Disease and Thalassemia Solid Tumors
Adolescent Hematology and Gynecology Brain and Spinal Cord Tumors Cell and Gene Therapy Epidemiology (Cancer and Hematology) ITP and Immune Hematology Liver Tumors Precision Oncology Sickle Cell Disease and Thalassemia Thyroid Tumors Bone Marrow Failure Cancer Genetics and Genomics Cell Therapy and Bone Marrow Transplant Hemostasis and Thrombosis Immunotherapy Lymphoma Rare Tumors Soft Tissue Sarcoma Bone Tumors Cancer Survivorship Developmental Therapeutics Histiocytosis Leukemia Neuroblastoma Retinoblastoma Solid Tumors

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