Final Research Report

View this project's final research report.

Peer-Review Summary

Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.

The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments. 

Peer reviewers commented, and the researchers made changes or provided responses. The comments and responses included the following:

  • The reviewers asked that the report give more attention to the difficulty of obtaining sufficient data from subjects for this study, noting that the reminder intervention seems a positive tool.  However, they explained that the mobile directly observed therapy (MDOT), requiring subjects to submit videos when taking medication, raised issues of privacy, ease of use, and effectiveness. The researchers added discussion to the report about the limitations of the study’s approach and the intrusiveness of MDOT. They noted that adherence to the study protocol of submitting videos may underestimate the participants’ adherence in taking hydroxyurea (HU) therapy because participants did not always send a video when they took HU.
  • Reviewers suggested that reframing the research as a mixed-methods study would be beneficial. The reviewers explained that in this type of study, the qualitative findings could better enhance the quantitative results, which the sample size limits. The researchers explained that while they reached thematic saturation in the qualitative analysis, it concerned them that this would be lost when conducting quantitative subgroup analyses, and they preferred to keep the analyses separate.
  • Reviewers noted that different types of sickle cell disease (SCD) produce different challenges and needs. They wondered if more insights would have been gleaned if this study identified patients by type of SCD. The researchers agreed that patients with different forms of SCD might have different responses to HU but said that this study did not have the statistical power to distinguish impact in patients with different types of SCD.
  • Reviewers noted that the rate of video submission seemed poor, and that the researchers’ use a rate of 25 percent adherence cut point to categorize groups seemed too low. The researchers acknowledged that they did not set a priori an adequate level of adherence for video submission, since there were no previous standards for this.  They explained that 25 percent was much lower that what previous research has set as a benchmark for good medication intake adherence (e.g., 80 percent). However, as they learned from qualitative interviews that participants often took medications but did not submit videos, they determined post hoc that the appropriate inflection point categorize groups was 25 percent adherence with video submission.

Conflict of Interest Disclosures

Project Information

Lakshmanan Krishnamurti, MD
Emory University^
$2,148,331
10.25302/05.2020.CE.13046859EM
Patient-Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy with Hydroxyurea in Patients with Sickle Cell Disease

Key Dates

September 2013
February 2019
2013
2018

Study Registration Information

^Lakshmanan Krishnamurti was affiliated with the University of Pittsburgh when this project was funded.

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Last updated: March 4, 2022