Hematologic Disorders

Hematologic disorders are conditions found in the blood and blood forming organs. This includes blood cell cancers, genetic disorders, anemia, HIV related conditions, sickle cell disease, and complications do to chemotherapy treatment or transfusions.

Our hematology team leads clinical trials that evaluate effective treatments through medicines, compare new therapies against existing protocols, and study groups of patients with hematologic conditions to decrease pain and reduce patient’s dependency on current treatments.

A Comprehensive Program to Increase Sickle Cell Trait Knowledge and Awareness Among Parents of Young Children Identified by Newborn Screening

Protocol ID: SCTaware

Disease: Sickle Cell Trait

Who can participate?: Biological parents of young children (<3 years old) with SCT identified by NBS who are referred and present for in-person SCT education at NCH

Lead Researcher: Susan E. Creary
A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD)

Protocol ID: ATHN9

Disease: VWD

Who can participate?: Participants with severe Von Willebrand Disease with Type 3 VWD or VWF:RCo, VWF:GPlbM or VWF:Ag ≤30% of pooled normal control plasma on more than one occasion; Participants with clinically severe VWD as defined by VWF:RCo, VWF:GPlbM or VWF:Ag ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates; and Co-enrollment in the ATHNdataset.

Lead Researcher: Amy L. Dunn
A Phase 3 Study of Eltrombopag vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Protocol ID: ICON 3

Disease: Thrombocytopenia

Who can participate?:  Age: 1- <18 years. Newly diagnosed ITP (<3 months from diagnosis (first abnormal platelet count). Platelets <30 x109/L at screening

Lead Researcher: Melissa J. Rose

A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders

Protocol ID: PIDTC 6901

Disease: SCID Disorders

Who can participate?: SCID being treated with transplant or PEG-ADA or gene therapy

A Randomized, Open-Label, Active Controlled, Safety and Extrapolated Efficacy Study in Pediatric Subjects Requiring Anticoagulation for the Treatment of a Venous Thromboembolic Event

Protocol ID: Pfizer Apixaban B0661037

Disease: Thrombosis

Who can participate?: Children 3 months to <18 years of age with a minimum weight of 6 kg at the time of randomization. Presence of an index VTE which is confirmed by imaging.

Lead Researcher: Sarah H. O'Brien
Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders. ITP Consortium of North America (ICON) Genetic Research Sample Bank

Protocol ID: ATHENA 1

Disease: Bleeding Disorders

Who can participate?: All female participants cared for at HTCs who are participating in this project may be included.

Lead Researcher: Sarah H. O'Brien
Dose escalation study to characterize pharmacokinetics after oral admin of eltrombopag in pediatric patient with refractory, relapsed, or treatment naïve severe aplastic anemia or recurrent aplastic anemia

Protocol ID: Eltromopag

Disease: Hematology

Who can participate?: Age 1-18y; New diagnosed or relapsed AA; bone marrow 4weeks prior to treatment; BMT not an option; normal FISH chromosome.

Lead Researcher: Melissa J. Rose

Engaging Parents of Children with Sickle Cell Disease and their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)

Protocol ID: ENGAGE

Disease: Sickle Cell

Who can participate?: Ages 0 to 5 (inclusive) with SCD diagnosis

Lead Researcher: Susan E. Creary

Genetic and Biological Determinants of Immune Thrombocytopenia

Protocol ID: ICON Bank

Disease: Thrombocytopenia

Who can participate?: Patients diagnosed with immune thrombocytopenia (including Evans syndrome if ITP is present).

Lead Researcher: Melissa J. Rose

Genotypic and Phenotypic Analysis of Adolescents with Heavy Menstrual Bleeding and Low Von Willebrand Activity

Protocol ID: HMB & Low VWF H-39295

Disease: Menorrhagia

Who can participate?: Post-menarchal females < 21 years of age with HMB (defined as PBAC score >100)

Lead Researcher: Sarah H. O'Brien
Hydroxyurea Optimization through Precision Study (HOPS)

Protocol ID: HOPS

Disease: Sickle Cell Anemia

Who can participate?: Children with confirmed (HbSS, HbSD or Hbβ0-thalassemia) who are initiating hydroxyurea therapy, following a discussion with their local clinical team, will be eligible for this study.

Lead Researcher: Susan E. Creary

ISTH Database and DNA Biorespository for Congenital Antithrombin Deficiency- pilot/feasibility study

Protocol ID: ISTH Database

Disease: Thrombosis

Who can participate?:

Ages 0-21 years

AT deficiency confirmed

Lead Researcher: Riten Kumar, MD

Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the treatment and control of bleeding episodes, the efficacy and safety of rVMF in Elective and Emergency Surgeries, and the PK or rVWF in Children Diagnosed with Severe von Willebrand Disease

Protocol ID: Baxalta 071102 rVWF (Von Vendi)

Disease: Von Willebrand Disease

Who can participate?: 3 age cohorts:

  • Cohort 1: >12 to <18 years
  • Cohort 2: >6 to <12 years (after 6 subjects from Cohort 1)
  • Cohort 3: <6 years (after 6 subjects in Cohort 2)
  • Severe VWD

Lead Researcher: Amy L. Dunn

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease

Protocol ID: BMT CTN 1507

Disease: SCD

Who can participate?: Must be b/t  5 – 14 yrs and had a stroke. Must be  b/t 15 – 45yrs and meet additional protocol criterion. Have a donor who is a half match.

Lead Researcher: Hemalatha Geetharani Rangarajan

Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease

Protocol ID: B5201003- Rivipansel Extension

Disease: Sickle Cell

Who can participate?: ≥ 6 years of age.

Lead Researcher: Anthony D. Villella

Community Counts Surveillance study

Protocol ID: Community Counts

Disease: Multiple Bleeding Disorders

Who can participate?: Patients aged 12-22 with bleeding disorders

Lead Researcher: Amy L. Dunn, MD
Adolescents Presenting to Young Women’s Hematology Clinics with Heavy Menstrual Bleeding: A Prospective Cohort Study and Research Registry

Protocol ID: HMB Registry

Disease: Menorrhagia

Who can participate?: Ages < 21, patients who are new referrals to a Young Women Hematology Clinic or General Hematology Clinic for evaluation of HMB

Lead Researcher: Sarah H. O'Brien, MD
Sever Chronic Neutropenia International Registry Protocol

Protocol ID: SCN Registry

Disease: SCN

Who can participate?: Age greater than three months

Lead Researcher: Melissa J. Rose, DO