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IPIG 2025 | Biomarkers in the era of complement inhibition for PNH
Austin Kulasekararaj, MBBS, MD, MRCP, FRCPath, King’s College Hospital NHS Foundation Trust, London, UK, comments on the utility of biomarkers in the era of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), emphasizing the importance of simple and easily accessible biomarkers. Dr Kulasekararaj highlights lactate dehydrogenase (LDH) as a valuable biomarker, as well as reticulocyte count and unconjugated bilirubin levels, which can aid in monitoring patients switching from C5 inhibitors to proximal inhibitors. Clinical symptoms are also helpful, particularly for identifying cases of acute hemolysis or breakthrough hemolysis. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.
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Transcript
As a clinician, I tend to go a bit more around simple biomarkers which are routinely available, widely available, easily accessible, quicker results as well. So in that context, we are quite blessed in the context of PNH that we have lactate dehydrogenase (LDH) which is available in every nook and corner of the world so physicians can use it more frequently as well and use that as a good biomarker for any patient with PNH, be it proximal or terminal inhibition...
As a clinician, I tend to go a bit more around simple biomarkers which are routinely available, widely available, easily accessible, quicker results as well. So in that context, we are quite blessed in the context of PNH that we have lactate dehydrogenase (LDH) which is available in every nook and corner of the world so physicians can use it more frequently as well and use that as a good biomarker for any patient with PNH, be it proximal or terminal inhibition. Additionally, we know in patients switching from C5 inhibitors to proximal inhibitors, we do have monitoring of reticulocyte count and also bilirubin, i.e., unconjugated bilirubin levels, which could also be quite helpful. Additionally, clinical symptoms are quite useful in this context when patients have acute hemolysis or breakthrough hemolysis, where patients can present with the same symptoms they presented at diagnosis, i.e., dark urine or abdominal pain, which were quite debilitating at the outset as well. So collectively, clinical biomarkers are pretty useful along with a good clinical assessment of the patients, that you can decide whether a patient is stable or they are having episodes of breakthrough, both in the context of proximal and also terminal inhibition.
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Disclosures
Research support (to institution): Celgene/BMS, Novartis; Consultant: Samsung, Novo Nordisk, Alexion/AstraZeneca, Arrowhead Pharmaceuticals, Silence Therapeutics, AdaRx, Ono Pharma; Speaker’s fees: Alexion/AstraZeneca, Amgen, Celgene/BMS, Pfizer, Novartis, Ra Pharmaceuticals/UCB, Roche, Sobi, Janssen; Scientific advisory board/data monitoring committee: Alexion/Astra Zeneca, Apellis, Amgen, Agios, BioCryst, Celgene/BMS, Geron, Novartis, Pfizer, Regeneron, Roche, Sobi, Janssen, Samsung.
