Researchers set out to determine whether existing patient-reported outcome (PRO) measures are sufficient to assess the impact on the quality of life of patients with cold agglutinin disease (CAD) and proposed their own set of PRO measures, as presented at the 63rd American Society for Hematology Annual Meeting & Exposition.

The constellation of symptoms typically experienced by a patient with CAD includes shortness of breath, fatigue, and weakness. This can potentially severely impact the ability of patients to carry out day-to-day activities. 

Various PRO measures have been proposed to gauge the CAD disease burden, and Joly and colleagues decided to investigate whether they were adequate. They first conducted a literature review on PubMed of studies published between 2010 and 2020 on existing PRO measures used to assess the symptoms, impact, and quality of life of adult patients with CAD.

Continue Reading

Read more about CAD diagnosis 

They then compiled those measures into questions they could ask in an interview. They conducted interviews with 37 participants with either primary or secondary CAD. From these interviews, they discovered that the most frequently reported symptom was reactions to cold environments, such as having cold or numb hands/feet, skin discoloration, muscle aches, and headaches.

The greatest impact of CAD is on the participants’ ability to conduct day-to-day activities, such as work, household chores, and errands. CAD also negatively impacted patients’ ability to conduct enjoyable activities, such as gardening and exercise. 

“Following a literature review, no existing PRO measure was found to adequately address concepts deemed critical to CAD from the patient perspective,” Joly et al wrote. Their proposed new item set consisted of 11 parameters, such as those relating to fatigue, cold sensitivity, dyspnea, and the wearing of extra clothing. 

“Participants reported these items were comprehensive of their experiences with CAD, easy to understand, and would be relevant to gaining a better understanding of individuals’ experiences with CAD,” they concluded.


Joly F, Dasmahapatra P, Su J, DiBenedetti D, Kosa K, Hill Q. Development of a cold agglutinin disease-specific patient-reported outcome symptom measure. Poster presented at: 63rd American Society for Hematology Annual Meeting & Exposition; December 13, 2021.