Core Outcome Set for Sickle Cell Disease in Pregnancy

Sickle cell disease (SCD) is one of the most common hereditary hemoglobinopathies, accounting for approximately 28,600 deaths in 2010. Pregnancy in women with SCD is currently viewed more favorably; however, adverse pregnancy outcomes still occur, resulting in maternal or fetal morbidity and even mortality. Studies to date differ with respect to the outcomes they report, as do the definitions of these outcomes. This hampers the ability to compare, contrast, and combine these study results; a process particularly crucial in study areas, which focus on small populations, where the feasibility of accruing sample sizes necessary to properly power individual studies is sometimes an insurmountable challenge.

The objective of this project is to create a core outcome set for studies focusing on the course and management of SCD in pregnancy.


Principal Investigator:
Dr. Ann Kinga Malinowski
Assistant Professor, University of Toronto
Clinical Investigator, Lunenfeld-Tanenbaum Research Institute
Maternal-Fetal Medicine Specialist, Mt. Sinai Hospital, Toronto

Dr. Rohan D’Souza
Assistant Professor, University of Toronto
Maternal-Fetal Medicine Specialist, Mt. Sinai Hospital, Toronto

Dr. Nadine Shehata
Associate Professor, University of Toronto
Member, Institute of Health Policy, Management, and Evaluation, University of Toronto
Obstetric Haematologist, Mt. Sinai Hospital, Toronto
Head, Transfusion Medicine, Laboratory Medicine, and Pathology, Mt. Sinai Hospital, Toronto

Dr. Richard Ward
Associate Professor, University of Toronto
Director, RBC Disorders Program, Toronto General Hospital

Dr. Kevin Kuo
Assistant Professor, University of Toronto
Clinician-Investigator and Staff Hematologist,
RBC Disorders Program & Therapeutic Apheresis Program, Toronto General Hospital

Further Study Information

Current Stage: Ongoing
Date: January 2019 - January 2020
Funding source(s): Pending

Health Area

Disease Category: Pregnancy & childbirth, Blood disorders

Disease Name: Sickle Cell Disease

Target Population

Age Range: 16 - 55

Sex: Female

Nature of Intervention: Any

Stakeholders Involved

- Clinical experts
- Consumers (patients)
- Methodologists
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Researchers
- Service providers
- Service users

Study Type

- COS for clinical trials or clinical research


- Consensus meeting
- Delphi process
- Literature review
- Systematic review

A systematic review of RCTs and cohort studies will be undertaken to index all reported outcomes and their definitions. Health service user input will be sought to further complement the outcomes compiled through the systematic review. The Delphi methodology will then be utilized to reach consensus among stakeholders with regards to a core outcome set. The outcome set identified through the Delphi method will then be refined via a consensus meeting.