Evidence map›Paper›PMID 42583311›Full record

ArticleEJHaem2026

Anemia and Patient-Reported Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Real-World Observational Study.

Esther Natalie Oliva, Antonio M Risitano, Eros Di Bona, Alessandro Maria Vannucchi, Ilaria Maria Dellfino, Antonio De Vivo, Valentina Giai, Rosario Notaro, Giuseppe Iannì, Eloise Beggiato and 13 more

Abstract read
In one paragraph

Article in EJHaem, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

23 authors.

Esther Natalie OlivaHematology Department London North West University Healthcare NHS Trust London UK.ORCID https://orcid.org/0000-0001-9238-5734
Antonio M RisitanoHematology and BMT Unit AORN San Giuseppe Moscati Avellino Italy.
Eros Di BonaUOC Oncoematologia AULSS7 Pedemontana Vicenza Italy.
Alessandro Maria VannucchiSOD Ematologia Azienda Ospedaliera Universitaria Careggi di Firenze Firenze Italy.
Ilaria Maria DellfinoHematology Unit Grande Ospedale Metropolitano Bianchi Melacrino Morelli Reggio Calabria Italy.
Antonio De VivoInstitute of Hematology Bologna Italy.
Valentina GiaiHematology Department of Hematology and Oncology AOU Città della Salute e della Scienza di Torino Turin Italy.
Rosario NotaroSOD Ematologia Azienda Ospedaliera Universitaria Careggi di Firenze Firenze Italy.
Giuseppe IannìAssociazione QOL-ONE Reggio Calabria Italy.
Eloise BeggiatoHematology AOU Città della Salute e della Scienza di Torino-Presidio Ospedaliero Molinette Turin Italy.
Simona SicaDipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia Fondazione Policlinico Universitario A. Gemelli IRCCS Università Cattolica Sacro Cuore Rome Italy.
Gaetano La BarbaDepartment of Medicine and Aging Sciences University of Chieti-Pescara Pescara Italy.
Sneha BalakrishnanKing's College Hospital NHS Foundation Trust London UK.
Joanna LargeKing's College Hospital NHS Foundation Trust London UK.
Tanuja ThavarajahKing's College Hospital NHS Foundation Trust London UK.
Simona RasoDepartment of Hematology and Rare Diseases Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello Palermo Italy.
Raffaele FontanaHematology and Transplant Center University Hospital "San Giovanni di Dio e Ruggi d'Aragona" Salerno Italy.
Wilma BarcelliniS.C. Ematologia Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy.
Bruno FattizzoS.C. Ematologia Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy.
Roochi TrikhaDepartment of Medicine and Aging Sciences University of Chieti-Pescara Pescara Italy.
Luana MaranoHematology and BMT Unit AORN San Giuseppe Moscati Avellino Italy.
Jens PanseCenter for Integrated Oncology Aachen Bonne Cologne Düsseldorf (ABCD) Aachen Germany.
Austin G KulasekararajKing's College Hospital NHS Foundation Trust London UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by complement-mediated hemolysis, thrombosis, bone marrow failure, and anemia. Although current treatments improve outcomes, patients still experience hemolysis and could remain anemic with a negative impact on quality of life (QoL). The aim of this study was to assess whether patient-reported outcomes (PROs) distinguish PNH patients with/without hemolysis and evaluate factors associated with PROs. Methods: In this international, multicenter, observational study, 97 PNH patients witfrom Italy and the UK completed the EORTC QLQ-C30 and QLQ-AA/PNH measures at baseline and after 2 weeks. Clinical, biochemical, and treatment data were collected. Hemolysis was defined as LDH ≥ 1.5 × upper normal limit. PRO scores were compared between patients above and below this threshold. Associations of PROs with patient and disease factors were analyzed. Results: Of 97 patients (mean age 49 ± 15), 24.7% were anemic (Hb < 10 g/dL) and 16% had hemolysis. QoL did not differ significantly by hemolysis status. However, anemia was associated with significantly lower EORTC QLQ-C30 global health and functional scores ( Conclusions: Anemia, but not residual hemolysis, was associated with impaired QoL across multiple domains. Addressing anemia alongside hemolysis may improve PROs in the management of PNH. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.

Indexed as

EORTC QLQ‐C30hemolysisparoxysmal nocturnal hemoglobinuriapatient‐reported outcomesQLQ‐AA/PNHquality of life

Identifiers

PMID42583311
PMCPMC13460258

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.