Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804–11.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Schrezenmeier H, Röth A, Araten DJ, Kanakura Y, Larratt L, Shammo JM, et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505–14.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Hansen DL, Möller S, Andersen K, Gaist D, Frederiksen H. Increasing incidence and prevalence of acquired hemolytic anemias in Denmark, 1980–2016. Clin Epidemiol. 2020;12:497–508.

Article  PubMed  PubMed Central  Google Scholar 

Richards SJ, Painter D, Dickinson AJ, Griffin M, Munir T, Arnold L, et al. The incidence and prevalence of patients with paroxysmal nocturnal haemoglobinuria and aplastic anaemia PNH syndrome: A retrospective analysis of the UK’s population-based haematological malignancy research network 2004–2018. Eur J Haematol. 2021;107(2):211–8.

Article  PubMed  CAS  Google Scholar 

Jalbert JJ, Chaudhari U, Zhang H, Weyne J, Shammo JM. Epidemiology of PNH and real-world treatment patterns following an incident PNH diagnosis in the US. Blood. 2019;134(Supplement 1):3407.

Article  Google Scholar 

Hill A, Platts PJ, Smith A, Richards SJ, Cullen MJ, Hill QA, et al. The incidence and prevalence of paroxysmal nocturnal hemoglobinuria (PNH) and survival of patients in Yorkshire. Blood. 2006;108(11):985.

Article  Google Scholar 

Fattizzo B, Cavallaro F, Oliva EN, Barcellini W. Managing fatigue in patients with paroxysmal nocturnal hemoglobinuria: a patient-focused perspective. J Blood Med. 2022;13:327–35.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Devalet B, Mullier F, Chatelain B, Dogné JM, Chatelain C. Pathophysiology, diagnosis, and treatment of paroxysmal nocturnal hemoglobinuria: a review. Eur J Haematol. 2015;95(3):190–8.

Article  PubMed  Google Scholar 

Brodsky RA. Advances in the diagnosis and therapy of paroxysmal nocturnal hemoglobinuria. Blood Rev. 2008;22(2):65–74.

Article  PubMed  CAS  Google Scholar 

Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233–43.

Article  PubMed  CAS  Google Scholar 

Alexion Pharmaceuticals, Inc. Highlights of Prescribing Information: Soliris® (eculizumab) injection, for intravenous use. Available from: https://alexion.com/Documents/Soliris_USPI.pdf.

Alexion Pharmaceuticals, Inc. Highlights of Prescribing Information: Ultomiris® (ravulizumab-cwvz) injection, for intravenous or subcutaneous use. Available from: https://alexion.com/documents/ultomiris_uspi.

Stern RM, Connell NT. Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria. Ther Adv Hematol. 2019;10:2040620719874728.

Article  PubMed  PubMed Central  CAS  Google Scholar 

US Food and Drug Administration. Prescribing information: Ultomiris (ravulizumab). 2018. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761108s023lbl.pdf.

Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4):1840–7.

Article  PubMed  CAS  Google Scholar 

Kulasekararaj AG, Griffin M, Langemeijer S, Usuki K, Kulagin A, Ogawa M, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022;109(3):205–14.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Tran JL, Kumar S, Eulogio R, Ramirez E, Foschini L, Juusola JL. PSY22 Pain management strategies and activity tracker utilization in a large-scale chronic pain study. Value in Health. 2019;22:S378.

Article  Google Scholar 

Ramirez E, Tran JL, Bradshaw B, Kumar S, Tai CG, Foschini L, et al. Longitudinal changes in activity and sleep behaviors from activity trackers are associated with change in depression severity. Ann Behav Med. 2019;53:S74.

Google Scholar 

Bradshaw B, Konty KJ, Ramirez E, Lee WN, Signorini A, Foschini L. Influenza surveillance using wearable mobile health devices. Online J Public Health Inform. 2019;11(1): e249.

PubMed Central  Google Scholar 

Yellen SB, Cella DF, Webster K, Blendowski C, Kaplan E. Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J Pain Symptom Manage. 1997;13(2):63–74.

Article  PubMed  CAS  Google Scholar 

Acaster S, Dickerhoof R, DeBusk K, Bernard K, Strauss W, Allen LF. Qualitative and quantitative validation of the FACIT-fatigue scale in iron deficiency anemia. Health Qual Life Outcomes. 2015;13:60.

Article  PubMed  PubMed Central  Google Scholar 

Chandran V, Bhella S, Schentag C, Gladman DD. Functional assessment of chronic illness therapy-fatigue scale is valid in patients with psoriatic arthritis. Ann Rheum Dis. 2007;66(7):936–9.

Article  PubMed  PubMed Central  Google Scholar 

Hays RD, Schalet BD, Spritzer KL, Cella D. Two-item PROMIS® global physical and mental health scales. J Patient Rep Outcomes. 2017;1(1):2.

Article  PubMed  PubMed Central  Google Scholar 

Yu L, Buysse DJ, Germain A, Moul DE, Stover A, Dodds NE, et al. Development of short forms from the PROMIS™ sleep disturbance and Sleep-Related Impairment item banks. Behav Sleep Med. 2011;10(1):6–24.

Article  PubMed  PubMed Central  Google Scholar 

Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. Pharmacoeconomics. 1993;4(5):353–65.

Article  PubMed  CAS  Google Scholar 

Schober P, Boer C, Schwarte LA. Correlation coefficients: appropriate use and interpretation. Anesth Analg. 2018;126(5):1763–8.

Article  PubMed  Google Scholar 

Montan I, Löwe B, Cella D, Mehnert A, Hinz A. General population norms for the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale. Value in Health. 2018;21(11):1313–21.

Article  PubMed  Google Scholar 

Northwestern University. PROMIS® Reference Populations. Available from: https://www.healthmeasures.net/score-and-interpret/interpret-scores/promis/reference-populations.

Tundia N, Hass S, Fuldeore M, Wang LL, Cavanaugh T, Boone J, et al. Validation and U.S. population norms of health-related productivity questionnaire. Value in Health. 2015;18(3):A24.

Article  Google Scholar 

Quer G, Gouda P, Galarnyk M, Topol EJ, Steinhubl SR. Inter- and intraindividual variability in daily resting heart rate and its associations with age, sex, sleep, BMI, and time of year: retrospective, longitudinal cohort study of 92,457 adults. PLoS ONE. 2020;15(2): e0227709.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Bohannon RW. Number of pedometer-assessed steps taken per day by adults: a descriptive meta-analysis. Phys Ther. 2007;87(12):1642–50.

Article  PubMed  Google Scholar 

Beattie Z, Oyang Y, Statan A, Ghoreyshi A, Pantelopoulos A, Russell A, et al. Estimation of sleep stages in a healthy adult population from optical plethysmography and accelerometer signals. Physiol Meas. 2017;38(11):1968–79.

Article  PubMed  CAS  Google Scholar 

Escalante CP, Chisolm S, Song J, Richardson M, Salkeld E, Aoki E, et al. Fatigue, symptom burden, and health-related quality of life in patients with myelodysplastic syndrome, aplastic anemia, and paroxysmal nocturnal hemoglobinuria. Cancer Med. 2019;8(2):543–53.

Article  PubMed  PubMed Central  Google Scholar 

Schrezenmeier H, Petra M, Gérard S, Jeffrey S, Alvaro U-I, Jaroslaw PM, et al. Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry. Haematologica. 2014;99(5):922–9.

Article  PubMed  PubMed Central  Google Scholar