Who, or what, are atypical HUS PROs: are these people, concepts, or initiatives?

In medical terminology PRO stands for ‘Patient Reported Outcomes’ and refers to comments made directly and in the patients’ own words, providing insight into patient experiences and viewpoints without interpretation by others. PROs capture what matters most to actual patients by offering their authentic and unfiltered version of meeting challenges and living lives that go beyond clinical settings surrounding aHUS diagnosis and treatment. In other words, PROs get to the heart of patient care and quality of life with patient–reported status in areas of physical and mental health but also impact on daily life or well-being aspects such as social concerns, treatment satisfaction, and disease burdens such as economic stressors. 

Whether terminonolgy appears as ‘patient centered care’ or ‘patient centric approaches’, what should stand out is an emphasis on patients, as they and their families are truly ‘atypical HUS PROs’. Patients and aHUS family caregivers should be considered as ‘pros’ or experts in disease treatment and management issues, for who else can provide first-hand knowledge about living with atypical HUS and what it’s like both during and after acute aHUS episodes? It’s vital that patients and family caregivers be included alongside those who offer dedicated care and medical advancements. They’re more widely recognized as credentialed aHUS professionals who are experts in aHUS/TMA, but together with patient views we can create a synergistic picture where the sum is greater than its parts. Since aHUS can occur with wide variations in event duration, severity, and organ damage, PROs must consider various scenarios and types of patient experiences. Development of aHUS PRO tools must include all aspects of care and daily life, with varied viewpoints of patients to include those who: are on dialysis, experience long-term aHUS versus short infrequent episodes, consider discontinuing treatment, had a transplant, are concerned about relapse, are genetically predisposed but without disease activity, had pregnancy-related aHUS, and many more groups.

While there’s always been interest in getting patient opinions, holistic PROs aren’t yet common in clinical practice, research, or clinical trials. There’s still a need to create methods and tools to collect meaningful but measurable patient-reported data which can help medical professionals, academics, and others gain insight into myriad aspects which affect aHUS patients. Unfortunately marketing and communication teams tend to ‘lump’ all rare disease patients as a group assumed to have similar characteristics, despite concise articles which describe reasons why aHUS isn’t like other rare disease populations and overviews which detail existing barriers to aHUS clinical trials. Two of the 5 sections of the Global aHUS Patients’ Research Agenda reflect patients’ interest in the clinical, psychological and social-economic impact of having aHUS. (6 of 15 items, sections 4 & 5). Patients and family caregivers want to, and can, help build brighter futures for those with this rare disease. 

Here’s a bevy of aHUS-specific content regarding why PROs are so important to defining and refining everything within the aHUS space, from developing new treatments to establishing best practice guidelines or ‘standards of care’ to improving the quality of life for patients.

PROs & the aHUS Global Patient RegistryHow are You Doing? (our 4 part series)

(1)  Patient Reported Outcomes

(2)- PRO reporting in practice

(3)-Improving PRO reporting

(4)-PRO data in use

aHUS and Clinical Trials

Being an aHUS Trialist

Breaking down Barriers in aHUS Clinical Trials

2020 aHUS Therapeutic Drug Landscape: Overview

aHUS Community

aHUS Patient Centricity

Atypical HUS:  Not Like the Others

Long Term aHUS Effects

The role of Positive Health in the Dutch approach to aHUS


Experiencing aHUS – Viewpoints & Variations

Atypical HUS Caregivers

Living with aHUS: What would you Say?

Patient Engagement

It’s time to Re-define aHUS Patient Engagement

Rare Disease Day 2022 – aHUS Patient Questions

aHUS Advocacy – Punching above our Weight

Specific Topics -PROs

aHUS Health, Well Being, and Work

aHUS Patient Fatigue

It’s not about my Rare Disease

Mental Health: aHUS community during the Pandemic

Eculizumab aHUS Treatment: PESaM

The role of Positive Health in the Dutch approach to aHUS

aHUS Patient Experiences: the Diagnostic Journey – 2021: 42 question global POLL (N=227) Results: 4 Reports https://bit.ly/aHUS2021pollData

For many more Articles & Resources, visit our 

Atypical HUS Resources page of our Info Centre