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Published on: March 14, 2017
Recruitment of infants with sickle cell anemia to a Phase III trial: data from the BABY HUG study
Lynn Wynn1, Scott Miller, Lane Faughnan
1St Jude Children's Research Hospital, Memphis, TN, United States. lynn.wynn@stjude.org
Insights
Recruiting for large clinical trials like BABY HUG requires approaching all eligible infants with sickle cell anemia. Avoiding biases in patient selection significantly improves enrollment success for hydroxyurea research.
Area of Science:
- Pediatric Hematology
- Clinical Trial Recruitment
- Sickle Cell Anemia Research
Background:
- Protocol-eligible subjects may decline research participation.
- Understanding factors influencing accrual is crucial for clinical trials.
- The BABY HUG trial investigated hydroxyurea in infants with sickle cell anemia.
Purpose of the Study:
- To determine factors affecting subject enrollment in the BABY HUG trial.
- To evaluate the impact of different recruitment strategies on trial participation.
Main Methods:
- Utilized an anonymized registry of potential subjects for data collection.
- Included infants under 18 months with hemoglobin FS diagnosis.
- Collected data on subject eligibility, parental approach, and participation reasons.
Main Results:
- 28% of potential participants were not approached due to reasons like poor adherence.
- Key participation drivers included contributing to knowledge, hope for hydroxyurea, and closer care.
- Declining reasons included fear of research, transportation, and study demands.
- 234 (21%) subjects provided informed consent; enrollment correlated with the number of families approached.
- Excluding patients based on biases did not improve recruitment success.
Conclusions:
- Large clinical trials necessitate a substantial pool of potential participants.
- Approaching all eligible patients without bias is key to successful recruitment.
- Effective recruitment strategies are vital for advancing sickle cell anemia research.
Background:
Protocol-eligible subjects may not be candidates for research participation or may decline. To determine factors that affected accrual, we evaluated enrollment in BABY HUG, a multi-center, randomized, placebo-controlled Phase III trial of hydroxyurea (HU) in infants with sickle cell anemia.
Methods:
An anonymized registry of potential subjects served as the primary source of data. Study coordinators considered all infants less than age 18 months with a hemoglobin FS diagnosis on newborn screening. Data included the number of potentially eligible subjects, whether parents were approached, and reasons for participating or declining.
Results:
Of 1106 potential participants, 28% were not approached for reasons such as prior poor adherence to clinical care. Interested families expressed willingness to contribute to medical knowledge (51%), hope of being randomized to receive hydroxyurea (51%), and desire for closer clinical care (51%) as reasons for participating. Disease severity or the perception that their child was ill had less impact on willingness to participate (16%). Parents who declined cited fear of research (19%), transportation problems (14%), and the demanding nature of the study (25%). Ultimately, 234 (21%) gave informed consent, with little variability of acceptance rates among sites. Importantly, the number of subjects enrolled correlated with the number of families that were approached. Sites that excluded patients based on clinical/psychosocial biases were not more successful in recruiting than those who approached all eligible subjects.
Conclusion:
Large, demanding clinical trials require an adequate pool of potential participants. Approaching all potentially eligible patients without predetermined biases enhances success in recruitment.
