HRQL refers to either specific or global measures of subjective well-being. Tools for HRQL assessment can be useful in predicting outcomes, tailoring treatment and encouraging patient self-management of disease [
8]. In addition to HRQL, patient-reported outcomes or PROs have become increasingly common in use for clinical care and tap similar domains as specific HRQL tools. PROs have been defined as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.”. These tools enable assessment of patient–reported health status for physical, mental and social well–being, a variant of an HRQL measure. A wide variety of patient-level instruments to measure PROs have been used for clinical research purposes. Many of these have been evaluated and cataloged within NIH’s Patient Reported Outcomes Measurement Information System (PROMIS). While PROMIS and other initiatives have validated patient-level outcome measures and instruments, there are two major challenges to using them for purposes of accountability and performance improvement:
1.
They are not in widespread use in clinical practice.
2.
Little is known about aggregating these patient-level outcomes for measuring the performance of the healthcare entity delivering care [
9].
Early prototypes of PROs used by the authors, like patient symptom ratings, coupled with our provider rating tools, have aided in establishing a more holistic understanding of gastroparesis patients’ sense of well-being and the interruption of that sense of well-being due to complications related to gastroparesis. Particularly, our work has always included patient ratings (pre-PROs), as well as provider ratings. We contend that this mirror assessment offers a more comprehensive picture of the patient experience and provider view and lends itself to promoting both informed and activated patients and providers who feel more self-efficacy: the patient-provider partnership now advocated by the Institute for Healthcare Improvement and others [
10]. Lastly, the development of defined and reliable PROs for use in Gp patients has been recommended by the CDER [
6], who conclude that ideal PRO tools are lacking currently:
“Because gastroparesis is a symptomatic condition, a well-defined and reliable PRO instrument that measures all the clinically important signs and symptoms of gastroparesis would be the ideal primary efficacy assessment tool in clinical trials used to support labeling claims for the treatment of gastroparesis. However, at the current time, we know of no measure of clinically important gastroparesis signs and symptoms that would serve as the ideal primary efficacy assessment tool. Until an appropriate PRO instrument for gastroparesis becomes available, sponsors should consider the strategies discussed in the following sections when designing gastroparesis clinical trials. Sponsors may wish to explore new PRO instruments or novel diagnostic measures in early development, and potentially correlate the results with dose-ranging trials.”
We believe that the rating tools developed and described below constitute “new PRO instruments or novel diagnostic measures in early development” suggested by the CDER [
6].
Methodologies to determine the best patient care for people suffering from gastroparesis continued to be developed and refined by the corresponding author and his teams in the late 1980s through the 1990s. Along with the PRO/TSS, early work with patient ratings included a Symptoms Interview based on the severity and frequency of symptoms and the Short Form – 36 Version 1, a well-validated and psychometrically sound Health Related Quality of Life Measure. Two authors (TC and TA) then developed a provider-rating tool which was initially called A Diagnostic And Prognostic Score (or ADAPS) and later called the Investigator Derived Independent Outcomes Measure Score or IDIOMS. Essentially the ADAPS/IDIOMS tool was developed around 1995 to evaluate
three specific areas of patient experience, based on what patients named as important to them: (1) what they wanted to be addressed medically; (2) how sick they were with GI symptoms other medical problems, and; (3) what services they needed, a gross healthcare utilization measure. These areas correspond to healthcare utilization domains used as components of the Diagnostic Related Groups (DRGs) that the Center for Medicare & Medicaid Services (CMS) uses to set reimbursement rates [
12]. The GI specific provider rating or IDIOMS was also incorporated to determine its usefulness as a synergistic tool in cooperation with patient reported symptoms.
In the ADAPS/IDIOMS tool (Additional file
1) each of these three domains is assessed on a 1–5 scale and a total summed score, ranging from 1 to 30. Patient symptom ratings (termed total symptom scores or TSS), the IDIOMS and its earlier versions (e.g., the ADAPS) continually have been used by our GI motility team, in several locations, since 1995.
Support group therapy/Focus groups
From April 1990 to April 2002, support group sessions were held for GI motility disorder patients at University of Tennessee Health Science Center. These sessions occurred monthly (except for 4 months), then staggered to quarterly for the last 2 years, for a total of about 120 sessions. Although detailed demographic records were not kept, over 1000 non-unique persons attended these groups over the 12-year period; attendance ranged from 5 to 45 patients plus additional GI academic and clinical staff per session. The group was started in part because the behavioral management strategies for GI symptoms used at that time (mostly to cope with musculoskeletal pain) did very little to aid GI motility disorder patients, and the team wished to offer other self-management strategies to help patients coping with these difficult illnesses. There were few medical options for GI motility patients; gastric electrical stimulation, now used in some drug refractory patients, was still investigational. Comparisons between early support group attendees and non-attendees, matched on illness acuity, disability and duration, suggested that attendees required fewer pain medication prescriptions, made fewer calls and requests of their providers, and used fewer outpatient healthcare resources, compared to non-attendees [
8].
Support group format consisted of introductions (participants briefly telling their stories), followed by a professional speaker, and a subsequent question and answer period. Support group sessions lasted around 90 min, but often participants stayed for over 120 min. Focus group topics included pain management, partnering with your medical team, nutritional issues, total parenteral nutrition (TPN), family dynamics in chronic illness, navigating the insurance system, physical activity to improve motility function, coping with cyclic vomiting syndrome for adults and children, financial management when dealing with a chronic illness, autonomic nervous system function impact on GI symptoms, diabetes and GI motility disorder symptoms, coping with depression and anxiety, biofeedback for pain management, anger management, GI stimulation (pacing), dealing with social isolation secondary to nausea, vomiting, as well as explosive diarrhea and bloating.
Many patients found an understanding venue in which to share, often resulting in crying, anger or other strong negative emotions, but patients also laughed together. The group had many “inside” jokes and prided themselves on being able to laugh about vomiting and other socially unacceptable behaviors. Unilaterally, group members found that the group helped instill hope, as well as bolster their morale and courage in coping with Gp [
8]. However, because the group was hospital-based and many of the more severely ill patients attended (with IV poles and in hospital gowns), some early outpatient attendees reported that seeing other patients with like illnesses who were so critically ill could be demoralizing. Some outpatients reported increased anxiety upon hearing about the rapid trajectory of illness severity and impairment for their hospitalized peers. Additionally, prior misdiagnoses and psychiatric overlay often placed upon patients (especially in rural areas with few specialists), generated many stories that shared how these experiences contributed to their pain and suffering. While all were allowed to share their stories and empathy was offered, the group focused on how to partner with healthcare professionals, versus feeling victimized by unintended iatrogenic impact from the medical and insurance systems.
The group also provided a forum for families and other patients to honor and memorialize those who died, and was a good venue for grief counseling for many families, once the family was ready to share about their loss. Unfortunately, many patients in our cohort (roughly 25 % from the initial University of Tennessee patient group) died in the course of those 12 years, with an 11.3 % mortality rate reported for a cohort of 214 who were treated with gastric electrical stimulation [
13].
Additionally, the group often shared their complementary medicine modalities with one another and joked about writing a “GI Motility Handbook of Home Remedies.” Interesting home-based modalities included eating Vietnamese soup and greasy Krystal burgers or having a family member use a plunger to “suction” the patients’ back or side when bloating was painful, etc., all offering some measure of relief for some patients. The group facilitator did not promote these measures as alternative medicines or substitutes for allopathic medicine, and always urged patients to consult with the GI motility team.
These support groups provided a good venue for building team partnerships with families and patients to improve treatment and communication, before patient and family-centered care or activated patient concepts were considered important or even named [
5]. Anecdotal information learned from patients about the experience of living with these disorders, as well as the impact on family life and coping were useful in creating/directing holistic treatment plans. Sharing from patients in these support groups helped the authors develop tools that combined health related quality of life measures or “pre-PRO” assessments, as ways to better capture and integrate the patient experience into treatment.