Mandy Nikpour answers the Holy Grail

Wednesday, 16 Sep 2015

Mandy Nikpour 5

In this week’s Holy Grail Mandy Nikpour from St Vincent’s Hospital in Melbourne tells us about her search for clinically important outcomes in scleroderma and lupus, and how she would like to answer one particular “million dollar question”.

What’s the issue your research is trying to solve?

My research is aimed at determining risk factors for clinically important outcomes in scleroderma and lupus, and working out strategies to modify these risk factors in order to improve outcomes.

What have you discovered so far?

My collaborators and I have developed a new model, based on blood NT-proBNP level and pulmonary function tests, to screen for pulmonary arterial hypertension (PAH) in scleroderma. This in turn enables earlier commencement of life-saving treatment for this serious and potentially lethal complication of the disease. We have also found that anticoagulation may be an effective treatment in scleroderma PAH.

We are now testing this hypothesis, which was generated in our observational cohort study, in a multi-centre clinical trial of the novel oral anticoagulant apixaban as adjunct therapy in scleroderma PAH. This study, which is NHMRC funded, is now under way in 13 centres across Australia.

What’s been your biggest hurdle?

I have been incredibly fortunate to date, to find like-minded collaborators willing to invest time and energy into answering the clinically important questions that really matter to our patients. Securing funding for our clinical trial from such a competitive source as NHMRC was a huge achievement as was getting our trial off the ground. However, our biggest hurdle, which is recruiting all the patients needed for our trial, lies ahead of us.

How far is your work from impacting patient care?

Being a clinical researcher, almost all of my work is intended to have a near-term impact on patient care. The development of a screening algorithm for scleroderma PAH, finding risk factors that identify patients with scleroderma interstitial lung disease who require aggressive therapy, and defining a low disease activity state that can serve as a treatment target in lupus are all examples of ways in which our research has impacted care.

If you could discover one thing in your research, what would it be? (e.g what’s your holy grail?)

I would love to see our clinical trial answer the million dollar question of whether patients with scleroderma PAH should be anticoagulated. It would be great if we could show that this treatment is safe and beneficial. We have to wait a few more years to know the answer, but hopefully the outcome will be worth the wait and all the hard work that we have put in!


Dr Mandana (Mandy) Nikpour, MBBS FRACP FRCPA PhD, is a physician-researcher at the University of Melbourne and St. Vincent’s Hospital Melbourne, with a clinical and research focus in systemic autoimmune disease, particularly systemic sclerosis (SSc; ‘scleroderma’) and systemic lupus erythematosus (SLE). She has extensive training and experience in study design and statistical analysis, and her methodological expertise lie in clinical measurement including the development of clinical tools for screening, prediction and outcome assessment, and also the design of clinical trials. She is CIA on a multi-centre RCT funded by an NHMRC project grant to evaluate the efficacy and safety of oral anticoagulation in SSc pulmonary arterial hypertension. Dr Nikpour currently holds an NHMRC Clinical Research Fellowship. She is co-Chair of the Damage Index Working Group of the International Scleroderma Clinical Trials Consortium. Dr Nikpour and her team have been awarded several prizes both nationally and internationally for their research.

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