How are you doing?(4)-PRO data in use

In the previous parts of this series “How are you doing?” the importance of PRO data , the level of compliance with PRO reporting in the Alexion aHUS Registry  and ideas for improving compliance have been discussed. In this final part an example of how PRO data can be used for research is presented,

As far as aHUS Global action is aware PRO data has only featured in one Alexion aHUS Registry research project*. It is about Fatigue and aHUS. And only an abstract has appeared.

The abstract can be read HERE.

It used Facit-Fatique PRO data from the aHUS  Registry that had been collected from adult patients on their enrollment up to the 5 May 2017.  Their health states at enrollment would be variable  and their PROs would reflect that.

651 adults had evaluable data. Others were either not adults or more relevantly  had not been supplied a PRO.

It  is known from Table 1 in Part 2 of this series  that approaching 300 did not compete their Fatigue PRO in their year of enrollment. Although some would be children, possibly 150 were adults.

So potential data was missing for this study from the outset

There are thirteen questions in the questionnaire, with five responses for each. Each reponse from   “not at all” which scores zero to “very much” which scores four is scored . Scoring then becomes  complicated process. The maximum score however  is 52.

The results revealed that at their time of enrollment , adult aHUS patients had an average Facit -fatigue score of 34.5 , signicantly  much less than the  maximum.

But the general population has a Facit-fatigue score of only 44. Still quite a gap to get to back to “normal”

34.5 was an average for all adult patients, some of whom were on dialysis at the time on enrollment , some with recurring TMA and others with neither. Some treated with plasma therapy and others receiving eculizumab.

Those aHUS patients still on dialysis had the lowest  Facit-fatigue score of  25.8 less than half of a maximum score.

Some were still experiencing TMA but scored 32.5.

Those with both resolved for more than 6 months  scored 38, but were still in the early period  of recovery.

The highest score in the whole range was 47.1  which is above general population average. aHUS patients can recover.

Although the results of the categories of patients can be compared with each other,  as well as with  the general population, in time, the groups can be self compared with future PRO results.

That needs those patients to continue to complete their questionnaires regularly for the rest of their time as participants in the Registry.

Although some  10 months after it appeared in the public domain, as it took time for aHUS Global Action to come across the abstract,  an article about this study appeared on the  aHUS Global Action  website, Global Action wrote :

This  is  just the base figure report and future analyses by the Registry will examine for any changes over time .

The quality and validity of follow up data depends on aHUS patients enrolled in the Registry completing their reports.

Their participation helps answer  questions  for others. 

aHUS patients included on-going ailments and side effects of aHUS and its treatment in their rearch agenda.

Facit-fatigue is one such ailment/side effect.

As time goes by and more observations are made then impact of recovery time and medical (e.g resolving anaemia) and other interventions ( e.g general fitness programmes)  could reveal an improvement in patients. If so, advice about interventions that work can be given to all aHUS patients for everyone’s benefit.

That would be the value that those participating in Alexion’s aHUS Registy and completing their PROs  could add to help all aHUS patients.

To go to Part 1 click HERE   Part 2 click HERE  Part 3 click HERE

*Facit fatigue results did appear in a general article about eculizumab safety using data from the Alexion aHUS Registry by the Rondeau Group these figures were presented

FACIT-Fatiguec score at baseline, median 30.0 (n = 59) 38.0 (n = 192) 33.0 (n = 12) 46.5 (n = 100)

Of interest is the sample size on which the FACIT Fatigue calculations are based ( i.e. n= ). The total sample numbers respectively of adults and children , ever or never treated with eculizumab , from which FACIT fatigue samples relate were 535, 307 and 330,149 respectively

The author would like to acknowledge Imad Al-Dakkak of Alexion for providing the data and Linda Burke for her help and suggestions on content .

Len Woodward , the author , has received travelling expenses and honoraria from Alexion in the past.