Last summer, aHUS alliance’s Twitter Account featured the plight of a patient who, after a week in hospital, had been found to have aHUS. By then the patient was in end stage kidney failure and on dialysis. On Twitter his family was seeking a matched kidney donor for him to have a kidney transplant.
The aHUS patient lives in the USA, and in Boston, the home of Astra Zeneca’s Business Unit for Rare Diseases, and it was 2021, drugs for treatment have been available for years.
There is no doubt that the world of aHUS has progressed significantly in the past decade or so. There has been improved awareness and treatment. The high cost of that treatment has in itself created an awareness, not just of aHUS unmet needs, but also those of people with rare diseases.
Access to aHUS treatment remains problematical for the majority of aHUS patients around the world this year. If they were all getting access for the whole year, sales by Astra Zeneca’s Business Unit for Rare Diseases sales would probably grow by around $2 billion dollars from aHUS alone. Its sales are growing but not at that rate.
Only some in the USA, and in parts of Europe, and Japan are now accessing ravulizumab treatment.
But access to treatment is not the only issue facing aHUS patients. For treatment to work sufficiently for the patient’s health, aHUS has to be recognised by clinicians in a timely manner.
Like all manner of statistics relating to global aHUS no one knows how many times aHUS has not been recognised in time to benefit the patient. But it has happened , as the USA patient on Twitter’s experience no doubt testifies.
As well as an unrivalled aHUS treatment policy, the UK has a world recognised aHUS expert centre in Newcastle upon Tyne,
The UK National Health Service embraces transparency of care performance. There is an annual audit of the expert centre’s performance. The Audit is preceded by an Annual Report published by the National Renal Complement Treatment Centre. The Report provides a comprehensive analysis of the work done by the Centre and measures of its performance. ( Copies of the past years’ reports can be read on its website at this link HERE.)
Within the Report, the Centre discloses performance data for several care areas, including new onset diagnosis and treatment,as well prevalent patients awaiting transplants.
Table 1 gives information accessed in the reports for the past five years on patients who have received an aHUS working diagnosis and eculizumab treatment, how some experience no renal improvement and how many have their working diagnosis revised leaving the remainder added to longer term eculizumab treatment list.
| Reporting Period | aHUS working diagnosis | No renal Improvement | Revised diagnosis/withdrawn | Net treated patients | Total ongoing treated | |
| 7/2016 to 3/2017 | 32 | -3 | -9 | 20 | 121 | |
| 4/2017 to 3/2018 | 38 | -10 | -11 | 17 | 114* | |
| 4/2018 to 3/2019 | 36 | -11 | -9 | 16 | 154* | |
| 4/2019 to 3/2020 | 36 | -13 | -8 | 15 | 125* | |
| 4/2020 to 3/2021 | 22 | -9 | -8 | 5 | 130* |
Table 2 shows the movement on aHUS patients on dialysis waiting for a transplant.
Table 2 aHUS Dialysis patients listed for transplant
| Reporting period | Carried forward from previous period | Transplanted patients | Newly listed for transplant | Listed at end of reporting period | |
| 7/2016 to 3/2017 | 30 | -3 | +5 | 28 | |
| 4/2017 to 3/2018 | 28 | -3 | +4 | 23* | |
| 4/2018 to 3/2019 | 23 | -5 | + 3 | 21 | |
| 4/2019 to 3/2020* | 21 | -4 | +7 | 24 | |
| 4/2020* to 3/2021 | 24 | -5 | +4 | 26* |
*as at 31 March 2021
Note: Of 221 patients referred to the centre in year 2020/2021 199 were not recommended to have eculizumab. 20 (10.1%) were subsequently found to have aHUS susceptiblity mutations and identified as patients who might benefit with eculizumab in the future, e.g at time of transplantation. In the previous four years fewer such patients were found between 4 and 5%.
Everyone knows that most dialysis patients may wait three years or more for a suitable donor to be found. Living donors and altruistic donation may reduce that wait. 10 to 25% aHUS patients may be chosen for transplant each year. But the number still waiting reduces only a little as new aHUS patients with renal failure are added to the list.
This is likely to be the situation in other countries with a met needs for complement inhibition drugs. In the USA there be more than 120 aHUS patients waiting for a kidney transplant, just like the Twitter donor appeal story patient. It is likely in 2022 that another 30 more new aHUS will not be diagnosed and treated in time.
So there is still some way to go yet to diagnose and get treatment in time to patients when needed.
We are not there yet.
Article No. 481
