Pediatric Blood Diseases
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Overview
The Children's Cancer and Blood Disease Program at UCSF Benioff Children's Hospital is an international leader in the treatment of cancer and blood diseases. We are home to one of the nation's 10 comprehensive Sickle Cell Centers and a regional pediatric Hemophilia Treatment Center. The Bone Marrow Transplant Program at UCSF Benioff Children's Hospital is a leader in special treatment options for children with primary immunodeficiency diseases, marrow failure syndromes, genetic diseases, cancers and other life-threatening illnesses.
UCSF Benioff Children's Hospitals medical specialists have reviewed this information. It is for educational purposes only and is not intended to replace the advice of your child's doctor or other health care provider. We encourage you to discuss any questions or concerns you may have with your child's provider.
Awards & achievements
Best in Northern California and top 10 in the nation for cancer care
Ranked among the nation's best in 11 specialties
Clinical trials
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones TrialOpens in a new window
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-40 years old...Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)Opens in a new window
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell DiseaseOpens in a new window
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD)...The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell DiseaseOpens in a new window
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD)...
Research initiatives
New Approaches to Neuroblastoma Therapy ConsortiumOpens in a new window
NANT is an international consortium that runs early-phase clinical trials, translating laboratory discoveries into new precision therapies for children with high-risk or relapsed neuroblastoma.