What the trial was testing
The BABY HUG enrolled 193 patients with sickle cell disease. The study was sponsored by National Heart, Lung, and Blood Institute (NHLBI) and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Hydroxyurea reduced painful episodes by more than half in toddlers with sickle cell disease.
Lancet (London, England) · 2011 · NCT00006400
These findings — that children taking hydroxyurea had about half as many painful crises compared to those on placebo — were published in the Lancet (London, England) and represent the headline result of the study.
Researchers tracked outcomes across 193 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with sickle cell disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Hydroxyurea is FDA-approved for sickle cell disease and is now recommended for very young children based on this study. It significantly reduces pain crises and hand-foot swelling, though it doesn't prevent all organ damage. Talk to your child's doctor about whether hydroxyurea is right for them.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open sickle cell disease trials
Autologous Testicular Tissue Transplantation
Freezing testicular tissue of prepubertal boys is a method for preserving spermatogonial stem cells in case of imminent gonadotoxic treatment during childhood. In case of total azoospermia or absence of spermatozoa that can be used for intra-cytoplasmic injection (ICSI) in adulthood, the investigators intend to perform the first in men autologous testicular tissue transplantation to restore fertility.
Minimizing Toxicity in HLA-identical Sibling Donor Transplantation for Children With Sickle Cell Disease
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).