It is fashionable these days to talk about precision medicine and tailor made/ personalised medicine.
Whilst it is easy to say it is difficult to envisage in practice.
What does it mean when aHUS patients are among a small subset of all TMAs. And no one can say precisely what aHUS is or more importantly what a doctor may think it is.
In fact, not only is aHUS itself heterogeneous ( diverse), but clinicians have tended to use the nomenclature inconsistently. For example, one group of clinicians may use the term aHUS as it was used in the 1970s (anything that is not STEC HUS),8 whereas other clinicians use it to refer specifically to HUS driven by uncontrolled activation of the complement system.13 ( from NKF report on aHUS nomenclature)
And even if it were confined to the “uncontrolled activation of the complement system” driver, each patient would be scattered among a “matrix” of possibilities for a complement thrombotic microangiopathic event.
aHUS patients whether currently treated or not. Whether never or ever will be treated fall within the boundaries or this box below.
Today the prevalent population of 30,000 aHUS patients can be found in this box. Each a pixel of their own. Each defined by their predisposition to the disease and their exposure to an illness triggering event.
So what is the precise medicine they need and how will it be tailored to each and their individual needs. All 30,000 of them alive today and 4000 more to come each year.
Also each falls within a healthcare system which ranges from state of the art to barely rudimentary.
What they may need and what is available to them is very different. Even among the needs for the fortunate few their wants are having to be satisfied too if it is to be personalised.
At its core those with cTMA, if it does not self resolve, need some form of complement inhibition. Once that need is satisfied what they want is their preferred way of receiving the complement inhibitor secure in the knowledge that their complement is inhibited.
Some will always want their treatment even when they no longer need it.
| Need | Not Need | |
| Want | Accepted Life | Dependent Life |
| Not Want | Burdened Life | Normal Life |
Some need to be rid of all treatment and return to normality as soon as they can.
So we now have just two dimensions of precision and tailoring cTMA treatment the way it is wanted to be administered and time it is needed for doing its job.
And within the “matrix” box above we have an “environment ” which every individual aHUS / cTMA patient is positioned.
We are beginning to relate aHUS patients to at least one dimension of the definition of precision tailored medicine given below. i.e. treatment.
By recognising the differing health care environment we are also relating it to developed and developing world characteristics.
Precision medicine is a medical approach that tailors disease prevention, diagnosis, and treatment to an individual’s unique genetic, molecular, environmental, and lifestyle characteristics.
30,000 individual effective preventions, 30,000 individual accurate diagnoses. 30,000 individual needed treatments for each of 30,000 individual genetic predispositions and 30,000 individual molecular circumstances and 30,000 individual life style characteristics.
So easier to say than to actually achieve.
Adapting a well known theory of human needs, a Maslow Hierarchy of Needs for Precision, Tailored Medicine in cTMA is basically about some arriving at the pinnacle whilst for the many is it is about striving for life and security.
For the majority of patients they cannot even access the most basic treatments to keep them alive let alone benefit from a very expensive essential complement TMA treatment a complement inhibitor. Plasma exchange would be a luxury. Within it there will be a different treatment tailoring experience for the 16 or so new African aHUS patients onsetting in the next 7 days than it will be for the or 8 EU/USA new patients
Tailoring for those who need and those who do not by being precise about it would be an important step in what seems to be an impossible endeavour.
Precision medicine at each stage so multiply the 30.000 going through potentially six stages.
From surviving to thriving.
Article No. 828
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