OBJECTIVES

To identify global real-world data (RWD) sources available for the rare blood disease Paroxysmal Nocturnal Hemoglobinuria (PNH) in order to summarize and understand the availability of RWD in this rare disease.

METHODS

A literature review was conducted to identify RWD sources for PNH using Medline and EMBASE from inception until June 2019. Identified literature was reviewed and a list of unique data sources was derived. Data sources were examined and meta-data for 90 variables was extracted to describe details around type of data source, study design, population size, epidemiology, clinical, economic and humanistic burden, follow-up duration, data access and linkage.

RESULTS

A total of 657 identified publications were screened. Among these, 45 potential RWD sources were identified - 80% were generic and 20% disease specific. Almost one-third of the data sources covered Asia, followed by Europe (24%), Americas (20%) and Oceania (2%); 7% were multi-regional. The majority of the RWD sources were administrative. Approximately half of the data sources (51%) reported a sample size in the range of 101 to 500. Data sources recorded minimum 2% and maximum 61% of the 90 assessed variables. More than 60% of RWD sources reported age, gender, diagnosis, PNH clone size and incidence of thrombotic events; the least (<1%) reported variables were risk factors, caregiver involvement, cost and resource use. Lactate dehydrogenase (LDH) levels cut-off and hemoglobin levels were reported in 60% and 53% of data sources respectively.

CONCLUSIONS

It seems that there is only a limited collection of RWD sources available based on our current review. Thus, to close the gap in existing evidence in this disease area the importance of collaboration to leverage and generate relevant RWD in this rare disease needs to be highlighted.