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ASH 2024 | The association between anemia and patient-reported outcomes in the novel era of PNH treatment
Esther Oliva, MD, Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy, comments on the association between chronic hemolysis/anemia and patient-reported outcomes (PROs) in patients with paroxysmal nocturnal hemoglobinuria (PNH). Dr Oliva highlights that hemoglobin levels, rather than lactate dehydrogenase (LDH) thresholds, strongly correlate with PROs and a better quality of life. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript
Yes, so we assessed patients both in the UK in a single centre and in many centres in Italy, patient-reported outcomes to assess as a primary endpoint whether patients experiencing chronic hemolysis, whether they had worse outcomes, patient-perceived outcomes, compared to those that did not. So we used LDH threshold to differentiate the patients. We used the EORTC-QLQ-C30, which is the instrument that has been frequently used in PNH, and we wanted to evaluate also a new instrument, which is the QLQ-AA/PNH, which is specifically designed for PNH...
Yes, so we assessed patients both in the UK in a single centre and in many centres in Italy, patient-reported outcomes to assess as a primary endpoint whether patients experiencing chronic hemolysis, whether they had worse outcomes, patient-perceived outcomes, compared to those that did not. So we used LDH threshold to differentiate the patients. We used the EORTC-QLQ-C30, which is the instrument that has been frequently used in PNH, and we wanted to evaluate also a new instrument, which is the QLQ-AA/PNH, which is specifically designed for PNH. What we found was that LDH did not distinguish patients for patient-reported outcomes. However, there was a very strong correlation between hemoglobin levels and the patient-reported outcome measures. And only the QLQ, the PNH-specific instrument, for several domains was able to distinguish patients with more than 10 hemoglobin level. Again, we still have this threshold of 10, and less than 10 who perceived a better quality of life. So again, it’s a recommendation that patients should reach more than 10 hemoglobin level to live a better life.
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Disclosures
Grande Ospedale Metropolitano Bianchi Melacrino Morelli: Current Employment; Daiichi Sankyo: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria, Speakers Bureau; Janssen: Speakers Bureau; Halia Therapeutics: Patents & Royalties; Novartis: Consultancy, Honoraria, Speakers Bureau; Amgen: Consultancy, Honoraria, Speakers Bureau; Sobi: Consultancy, Honoraria, Speakers Bureau; Alexion: Consultancy, Honoraria, Speakers Bureau; Ryvu: Consultancy, Honoraria, Patents & Royalties.
