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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 and related immune-mediated blood disorders, collaborating with leading centers across the country to advance care for our patients.

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)—as a frontline treatment for children newly diagnosed with ITP who required medication but did not have severe bleeding.

The trial evaluated eltrombopag, a thrombopoietin receptor agonist already FDA-approved for chronic ITP, as a treatment for children with newly diagnosed immune thrombocytopenia who required pharmacological intervention but did not have severe bleeding. 

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 met early, the study was stopped at interim analysis and the results published in JAMA.

What This Means for Patients: Eltrombopag offers a meaningful, sustained response option for children with newly diagnosed ITP who need 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 hematologists hear from patients and families with ITP are: "What caused me to develop ITP?" and "Will my ITP go away?" 

To address this need, Texas Children’s investigators partnered to create the the ITP Consortium of North America (ICON) BioBank in 2015.

 Now the largest pediatric ITP biorepository in North America, it includes nearly 1,000 patients and collects DNA, RNA, and plasma across multiple disease timepoints.

This BioBank is housed at Texas Children's Hospital and operated in partnership with eight children's hospitals across the United States. The biobank collects DNA, RNA, and plasma at multiple time points throughout each patient's disease course, with the goal of unlocking more individualized and targeted treatments.

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

Our Impact: The ICON BioBank is enabling discoveries that connect 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 articles 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 pairing scientific leadership with national collaboration and patient‑focused education, we can ensure that discoveries translate to improved care.

Our Researchers

Brandon R. Lucari, MD

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Areas of Research in Cancer and Blood Disorders

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

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