In 1946, the Constitution of the World Health Organization defined health as “a state of complete physical, mental, and social well-being and not merely the absence of disease or infirmity.” Yet, until recently, medical care and health research have focused almost exclusively on physical disease-related outcomes. As medical innovations led to better treatment outcomes, the importance of assessing treatment protocols not only in terms of their physical effects but also in terms of the impact on quality of life (QOL) became apparent. The value of determining QOL of patients is now well recognized within health care practice and health research as beneficial in addition to standard clinical measures. For example, in adults with cataracts, QOL measures correlate more highly with patient-perceived disability than do standard clinical tests like visual acuity,
1,
2,
3 and so are being implemented in both clinical trials and clinical care.
Most patient reported outcome measures (PROMs), such as those available in the Patient Reported Outcomes Measurement Information System (PROMIS
®) developed by the US-based National Institutes of Health, are intended for use as primary or secondary end points in clinical studies of the effectiveness of treatment across a range of medical conditions.
4 In addition, PROMs can be used on an individual level to facilitate the design of treatment plans, and aid communication and management of chronic diseases. While the PROMIS
® instruments assess dimensions common to many acute and chronic diseases, including emotional distress, pain, physical function, and social roles/activities, they were not designed to be sensitive to the effects of ophthalmic conditions. Most PROMs are questionnaires composed of fixed sets of items, but some now use item banks and computer-adaptive testing
5,
6 to guide item selection based on the patient’s responses to prior questions, yielding more rapid and precise assessment.
Numerous questionnaires have been developed to evaluate the impact of specific ophthalmic conditions in adults. Adult onset cataracts in particular have been extensively researched, with at least 16 different PROMs available.
7 However, PROMs designed for adults treated for cataracts include activities that are not applicable to the pediatric population, including driving and work-related activities, while ignoring important pediatric domains such as development milestones, education, and bullying. Moreover, the intraoperative and postoperative adverse events, postoperative management challenges, and severity of visual impairment differ from those experienced by adults.
Many of the pediatric PROMs are generic tools designed to assess and compare health status among children with different diseases, among children undergoing alternative treatments for the same disease, or between sick and healthy children.
8,
9 Such PROMs are important to gain a more complete understanding of the burden of pediatric eye disease within the context of other pediatric diseases. On the other hand, generic pediatric PROMs may lack sensitivity to detect small but clinically significant differences in vision-related QOL over time or due to treatment in the context of clinical trials. Both pediatric vision-related PROMs
10,
11 and pediatric ophthalmic condition-specific PROMs
12,
13 have been developed, to evaluate vision-related activities, and mental and social well-being, outcomes that are difficult to quantify and are consistently underreported by physicians.
14Proxy reporting by a parent provides an opportunity to evaluate QOL when a child is too young or cognitively unable to complete a questionnaire or interview. However, the perception of the proxy is influenced by many factors, including whether the mother or father completes the questionnaire,
14 the age of parents,
15 if any siblings are disabled,
15 and parental QOL.
16 Parents consistently reported a lower QOL in their child than did the child in a recent study of low vision
17 and in a study of children undergoing cancer treatment.
18 For these reasons, the U.S. Food and Drug Administration currently discourages proxy-reported outcome measures.
19 The creation of pediatric selfreporting instruments which involve children in the development with the aim of capturing the child’s concerns and experiences directly has become a priority.
20,
21,
22There are currently a number of PROMs available that could be, or have been, used to assess children with cataracts, as shown in
Table 53.1. None were specifically
designed for children with cataracts, but they do address potential functional deficits due to the visual impairment or common comorbidities, such as amblyopia or strabismus. The table highlights the age range for which each PROM was designed, its subscales, and whether it is administered to the child or a proxy.
