Perceived benefits and risks of participation in a clinical trial for Ugandan children with sickle cell anemia

Pediatr Blood Cancer. 2020 Feb;67(2):e27830. doi: 10.1002/pbc.27830. Epub 2019 May 28.


Introduction: Understanding factors that affect the decisions of caregivers of African children to enroll their children in clinical trials would lead to more fully informed consent.

Methods: During the NOHARM study (NCT01976416), a placebo-controlled clinical trial of hydroxyurea for Ugandan children with sickle cell anemia (SCA), 206 caregivers were given a semistructured questionnaire about factors that influenced participation in the study and their perceptions of study benefits and risks. Factors were further assessed with focus group discussions.

Results: Caregivers identified education provided during the recruitment process (44%), the child's current poor state of health (35%), and the possibility of improvement in the child's health (16%) as their primary initial reasons for deciding to participate in the NOHARM trial. Concerns regarding the drug or participation in a research study, including the stated concern of death by several caregivers, were outweighed by the possibility of improvement in the child's health. During the study, 72% of caregivers cited improved health as an advantage of study participation, while disadvantages cited included the potential side effects of hydroxyurea, most of which did not occur during the trial.

Discussion: Our study findings highlight the generally poor state of health of Ugandan children with SCA, the desperation by caregivers for anything that could improve the child's health, and the inevitable improvements in care that result from strict adherence to a study protocol, even a protocol based on local guidelines. Studies in this vulnerable population must be careful not to portray improved care as a primary incentive for participation.

Keywords: Africa; clinical trial; enrollment; sickle cell anemia (SCA).

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Sickle Cell / drug therapy*
  • Anemia, Sickle Cell / pathology
  • Antisickling Agents / therapeutic use
  • Caregivers / psychology*
  • Child
  • Clinical Trials, Phase III as Topic
  • Decision Making*
  • Health Knowledge, Attitudes, Practice*
  • Humans
  • Hydroxyurea / therapeutic use
  • Parental Consent / statistics & numerical data*
  • Patient Participation / psychology*
  • Patient Participation / statistics & numerical data
  • Randomized Controlled Trials as Topic
  • Risk Assessment / methods*
  • Surveys and Questionnaires


  • Antisickling Agents
  • Hydroxyurea

Associated data