Maria‐Magdalena Balp

Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey

Bing Han, Maria‐Magdalena Balp, Josefin Snellman, Anggie Wiyani et autres

INTRODUCTION: Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare condition, characterised by complement-mediated red blood cell breakdown, resulting in haemolytic anaemia. Associated symptoms, such as fatigue, impact on patient quality of life. Little is known about the comparative perceptions of symptom burden and …

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Prospective validation of imaging and serum diagnostic biomarkers of steatohepatitis and fibrosis in MASLD: the LITMUS Imaging Study

Michael Pavlides, Yasaman Vali, Ferenc Emil Mózes, Kristy Wonders et autres

There is a need for robust evaluations of noninvasive biomarkers for metabolic dysfunction-associated steatotic liver disease. This prospective multicenter study assessed the diagnostic accuracy of imaging (including liver stiffness measurement (LSM) using magnetic resonance elastography (MRE) and FibroScan (vibration-controlled transient elastography (VCTE)), …

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Efficacy and Safety of Treatments for Paroxysmal Nocturnal Hemoglobinuria: A Systematic Literature Review

Shreyans Gandhi, Isobel Munro, Victoria Shodimu, Neil Webb et autres

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by complement-mediated hemolytic anemia and thrombosis. The first treatments approved were complement 5 inhibitors (C5is), eculizumab and ravulizumab. Recently approved treatments include pegcetacoplan, iptacopan, danicopan (as an add-on to a C5i), …

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Supplementary data: Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment

Austin Gladston Kulasekararaj, Phillip Scheinberg, Maria‐Magdalena Balp, Olivier Somenzi et autres

These are peer-reviewed supplementary materials for the article 'Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment' published in the Journal of Comparative Effectiveness ResearchThis study conducted an indirect treatment comparison …

Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment

Austin Gladston Kulasekararaj, Phillip Scheinberg, Maria‐Magdalena Balp, Olivier Somenzi et autres

This study conducted an indirect treatment comparison (ITC) of iptacopan, the first oral monotherapy targeting factor B, versus pegcetacoplan, a subcutaneous infusion targeting complement component 3, in patients with paroxysmal nocturnal hemoglobinuria (PNH) and residual anemia despite complement component 5 inhibitors (C5i). …

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Supplementary data: Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment

Austin Gladston Kulasekararaj, Phillip Scheinberg, Maria‐Magdalena Balp, Olivier Somenzi et autres

These are peer-reviewed supplementary materials for the article 'Indirect treatment comparison of iptacopan versus pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and residual anemia despite C5 inhibitor treatment' published in the Journal of Comparative Effectiveness ResearchThis study conducted an indirect treatment comparison …

Disease burden and treatment patterns of paroxysmal nocturnal hemoglobinuria in Japan: a real-world survey

Masatoshi Sakurai, Yasutaka Ueda, Naoshi Obara, Michel Kroes et autres

This study aimed to determine the clinical profile and disease burden of patients with paroxysmal nocturnal hemoglobinuria (PNH) in Japan using real-world data from the Adelphi Real World PNH Disease Specific Programme (DSP)™, a cross-sectional survey conducted between January and December 2022. …

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Clinical Profile, Humanistic and Economic Burden of Paroxysmal Nocturnal Hemoglobinuria in Patients Treated With C5 Inhibitors

S. Gandhi, Markus Philipp Radsak, Yasutaka Ueda, Maria‐Magdalena Balp et autres

Objectives: To evaluate disease burden among paroxysmal nocturnal hemoglobinuria (PNH) patients prescribed C5 inhibitors (C5i). Methods: Data were drawn from the Adelphi Real World PNH Disease Specific Programme, a cross-sectional survey of physicians and PNH patients in Australia, Canada, France, Germany, Italy, …

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CO50 Clinical Profile of Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Treated With Complement Inhibitors: Results From a Real-World Study

Alexander Röth, Maria‐Magdalena Balp, Anggie Wiyani, Jahnavi Yenamandra et autres

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PCR34 Burden of Transfusion in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): Results From a Real-World Study in European Countries

Talha Munir, Maria‐Magdalena Balp, Anggie Wiyani, Jahnavi Yenamandra et autres

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CO58 Comparative Efficacy of Iptacopan Monotherapy vs. Danicopan Add-on to C5i in Paroxysmal Nocturnal Hemoglobinuria (PNH) Results From an Indirect Treatment Comparison (ITC)

Phillip Scheinberg, Austin Gladston Kulasekararaj, Maria‐Magdalena Balp, Anggie Wiyani et autres

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HTA136 Evaluating the Value Elements Considered in Health Technology Assessments of Paroxysmal Nocturnal Hemoglobinuria Treatments: A Targeted Review

Anggie Wiyani, Misaki Okuyama, Eve McArthur, Maria‐Magdalena Balp

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