title: Development of a disease-specific quality of life questionnaire for patients with aplastic anemia and/or paroxysmal nocturnal hemoglobinuria (QLQ-AA/PNH)-report on phases I and II.
abstract: Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated ultra-rare diseases. Quality of life (QoL) evaluation tools used in studies for AA and PNH are unspecific and designed for cancer patients (e.g., the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, EORTC QLQ-C30). Given the complexity of AA and PNH, variation in symptoms and treatments, younger age of many patients, and the fact that AA and PNH are not classified as malignant diseases, it is likely that cancer-specific questionnaires are inappropriate. We generate an AA/PNH-specific QoL questionnaire (QLQ-AA/PNH), performed according to EORTC guidelines. QoL issues were obtained from the literature and interviews with patients and physicians (phase I), then ranked by patients and physicians. In phase II, items were created. Patients in more than 25 German and Swiss cities were interviewed face to face. In phase I, interviews of 19 patients and 8 physicians specialized in AA/PNH treatment resulted in 649 QoL issues; these were condensed to 175 and graded according to their importance by 30 patients and 14 physicians (phase II). Five physicians took part in phases I and II. Altogether, 97 issues were rated important. Twelve EORTC QLQ-C30 items were not rated important, while several new QoL aspects were brought up. Modifications in wording and phrasing led to two questionnaires with 77 items regarding general QoL aspects and 20 items regarding medical care. Important QoL aspects of PNH/AA patients are inappropriately captured with available QoL tools. Developing a new QoL questionnaire specific for this patient group is warranted.
authors.affiliation: Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Germany.
authors.forename: Martha
authors.initials: M
authors.lastname: Groth
authors.affiliation: Division of Epidemiology and Health Services Research, Institute of Medical Biostatistics, Epidemiology and Informatics, University Medical Centre, Mainz, Germany.
authors.forename: Susanne
authors.initials: S
authors.lastname: Singer
authors.affiliation: Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Germany.
authors.forename: Cathrin
authors.initials: C
authors.lastname: Niedeggen
authors.affiliation: Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Germany.
authors.forename: Andrea
authors.initials: A
authors.lastname: Petermann-Meyer
authors.affiliation: Department of Hematology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Röth
authors.affiliation: Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and Institute of Transfusion Medicine, University of Ulm, Ulm, Germany.
authors.forename: Hubert
authors.initials: H
authors.lastname: Schrezenmeier
authors.affiliation: Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and Institute of Transfusion Medicine, University of Ulm, Ulm, Germany.
authors.forename: Britta
authors.initials: B
authors.lastname: Höchsmann
authors.affiliation: Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Germany.
authors.forename: Tim H
authors.initials: TH
authors.lastname: Brümmendorf
authors.affiliation: Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, University Hospital RWTH Aachen, Pauwelsstr. 30, 52074, Aachen, Germany. jpanse@ukaachen.de.
authors.forename: Jens
authors.identifier: http://orcid.org/0000-0001-6316-3112
authors.initials: J
authors.lastname: Panse
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000175:diagnosis / Q000453:epidemiology* / Q000523:psychology*; D005260:Female; D006457:Hemoglobinuria, Paroxysmal / Q000175:diagnosis / Q000453:epidemiology* / Q000523:psychology*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011788:Quality of Life / Q000523:psychology*; D011795:Surveys and Questionnaires / Q000592:standards*; D055815:Young Adult
keywords: Aplastic anemia; Bone marrow failure syndromes; Paroxysmal nocturnal hemoglobinuria; Quality of life