Our expertise in sickle cell disease in children and young adults
The Sickle Cell Disease Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center provides diagnosis, treatment, and long-term health management for infants, children, adolescents, and young adults with sickle cell disease.
We are an international leader in improving treatment and quality of life for children affected by all types of sickle cell disease, including:
- Sickle cell anemia (HbSS)
- Sickle cell with hemoglobin C disease (HbSC)
- Hemoglobin S-beta-thalassemia
- Other less common compound forms of sickle cell disease, including HbSD, HbSO, and HbSE
Base editing leaves Branden feeling ‘more than fine’
Branden Baptiste was the first person in the world to receive base editing, a highly precise form of gene therapy, for sickle cell disease.
Supportive care for pediatric sickle cell disease
From the first visit, our team of professionals are focused on supporting the needs and overall health of babies, children, adolescents, and young adults with sickle cell disease, including:
- Preventive therapies that may help your child avoid symptoms
- How to recognize the early signs of complications
- Managing sickle cell pain crises
- Screening for the risk of stroke and other acute complications
We'll work with you to create a care plan that's best for your child, taking into account your child's age, overall health, and medical history; type of sickle cell disease and how it is expected to progress; and tolerance for specific medications, procedures, or therapies.
Expanding care options for sickle cell disease
Research, much of it done right here at Boston Children’s, has created treatment options for sickle cell disease, including stem cell transplantation and gene therapy.
Our specialized sickle cell disease treatment team
The sickle cell disease team at Dana-Farber/Boston Children's includes hematologists, nurse practitioners, and physician assistants with extensive expertise in treating sickle cell disease. Many of our physicians are also active clinical and laboratory researchers, so our patients have access to the most up-to-date treatments available.
Because sickle cell disease can affect many organs in the body, your care team may also include radiologists, pulmonologists, nephrologists, ophthalmologists, surgeons, and other specialists as needed.
All of our patients are assigned a social worker and meet with our sickle cell disease psychologist, who can help manage the challenges of ongoing treatment and provide emotional, psychological, and logistical support.
Education and resources
Our nurses will be on hand and available by phone to walk you through your child's treatment and help answer any questions you may have about sickle cell disease: How can I prevent complications or recognize them if they occur? How often will my child require follow-up? They also will reach out to you by phone, continuing the care and support you receive while at Boston Children's.
For more information on sickle cell disease and how to manage your child’s symptoms, download our sickle cell anemia family education packet (PDF).
Our sickle cell disease research
Dana-Farber/Boston Children’s Pediatric Sickle Cell Disease Program has long been grounded in scientific research, in the clinic and in our laboratories. We are among the first of 10 sickle cell centers in the U.S. funded by the National Institutes of Health, and we are a site for the Sickle Cell Disease Clinical Research Network.
In the 1980s, our physicians were the first to recognize the potential of hydroxyurea to treat sickle cell disease and to use the medication in children. More recently, they have developed stem cell transplantation and gene therapy as treatments for sickle cell disease.
In 2008, our scientists discovered a new approach: silencing a gene called BCL11A. Suppressing this gene allows people with sickle cell disease to produce a fetal form of hemoglobin, preventing their red blood cells from sickling and making their symptoms much milder. This work led to the approval of CASGEVY™ gene therapy for sickle cell disease in 2023.
Newer approaches we’re exploring include base editing, a highly precise form of gene therapy that we are testing in a clinical trial, and a potential pill that targets BCL11A, currently in the early stages of laboratory research.
