Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria-a pilot study (ePRO-AA-PNH)

Silas Bänziger, Kimmo Weisshaar, Reetta Arokoski, Sabine Gerull, Jörg Halter, Alicia Rovó, Mario Bargetzi, Jeroen S Goede, Yuliya Senft, Sabine Valenta, Jakob R Passweg, Beatrice Drexler, Silas Bänziger, Kimmo Weisshaar, Reetta Arokoski, Sabine Gerull, Jörg Halter, Alicia Rovó, Mario Bargetzi, Jeroen S Goede, Yuliya Senft, Sabine Valenta, Jakob R Passweg, Beatrice Drexler

Abstract

Introduction: Electronic patient-reported outcomes (ePRO) are increasingly recognized in health care, as they have been demonstrated to improve patient outcomes in cancer, but have been less studied in rare hematological diseases. The aim of this study was to develop and test the feasibility of an ePRO system specifically customized for aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH).

Methods: After performing a user-centered design evaluation an ePRO system for AA and PNH patients could be customized and the application was tested by patients and their medical teams for 6 months. Symptom-reporting triggered self-management advice for patients and prompts them to contact clinicians in case of severe symptoms, while the medical team received alerts of severe symptoms for patient care.

Results: All nine included patients showed a high adherence rate to the weekly symptom-reporting (72%) and reported high satisfaction. The system was rated high for usage, comprehensibility, and integration into daily life. Most patients (78%) would continue and all would recommend the application to other AA/PNH patients. Technical performance was rarely a barrier and healthcare providers saw ePRO-AA-PNH as a useful supplement, but the lacking integration into the hospital information system was identified as a major barrier to usage.

Conclusion: An ePRO system customized for AA and PNH was feasible in terms of adherence, satisfaction, and performance, showing a high potential for these rare conditions in terms of data collection and patient guidance. However, the integration into clinical workflows is crucial for further routine use.

Trial registration: ClinicalTrials.gov NCT04128943.

Keywords: Aplastic anemia; Electronic health record; Paroxysmal nocturnal hemoglobinuria; Patient-reported outcomes; Self-management; ePRO.

Conflict of interest statement

RA is an employee of Kaiku Health Ltd. The other authors declare no competing interests.

© 2022. The Author(s).

Figures

Fig. 1
Fig. 1
Application development. Steps of the application development until the feasibility study
Fig. 2
Fig. 2
Patient adherence to the weekly questionnaire over the 6-month study time
Fig. 3
Fig. 3
Reported symptoms. Grade 1 corresponds to mild and grade 2 to moderate symptoms, which both resulted in self-management advice for the patients, and grade 3 corresponds to severe symptoms, sending out additionally an alert to the patients and physicians

References

    1. Richards HS, Blazeby JM, Portal A, Harding R, Reed T, Lander T, et al. A real-time electronic symptom monitoring system for patients after discharge following surgery: a pilot study in cancer-related surgery. BMC Cancer. 2020;20(1):543. doi: 10.1186/s12885-020-07027-5.
    1. Bingham CO, 3rd, Gaich CL, DeLozier AM, Engstrom KD, Naegeli AN, de Bono S, et al. Use of daily electronic patient-reported outcome (PRO) diaries in randomized controlled trials for rheumatoid arthritis: rationale and implementation. Trials. 2019;20(1):182. doi: 10.1186/s13063-019-3272-0.
    1. Benze G, Nauck F, Alt-Epping B, Gianni G, Bauknecht T, Ettl J, et al. PROutine: a feasibility study assessing surveillance of electronic patient reported outcomes and adherence via smartphone app in advanced cancer. Ann Palliat Med. 2019;8(2):104–11. doi: 10.21037/apm.2017.07.05.
    1. Falchook AD, Tracton G, Stravers L, Fleming ME, Snavely AC, Noe JF, et al. Use of mobile device technology to continuously collect patient-reported symptoms during radiation therapy for head and neck cancer: a prospective feasibility study. Adv Radiat Oncol. 2016;1(2):115–121. doi: 10.1016/j.adro.2016.02.001.
    1. Judson TJ, Bennett AV, Rogak LJ, Sit L, Barz A, Kris MG, et al. Feasibility of long-term patient self-reporting of toxicities from home via the Internet during routine chemotherapy. J Clin Oncol. 2013;31(20):2580–2585. doi: 10.1200/JCO.2012.47.6804.
    1. Andikyan V, Rezk Y, Einstein MH, Gualtiere G, Leitao MM, Jr, Sonoda Y, et al. A prospective study of the feasibility and acceptability of a Web-based, electronic patient-reported outcome system in assessing patient recovery after major gynecologic cancer surgery. Gynecol Oncol. 2012;127(2):273–277. doi: 10.1016/j.ygyno.2012.07.124.
    1. Basch E, Deal AM, Kris MG, Scher HI, Hudis CA, Sabbatini P, et al. Symptom monitoring with patient-reported outcomes during routine cancer treatment: a randomized controlled trial. J Clin Oncol. 2016;34(6):557–565. doi: 10.1200/JCO.2015.63.0830.
    1. Denis F, Yossi S, Septans AL, Charron A, Voog E, Dupuis O, et al. Improving survival in patients treated for a lung cancer using self-evaluated symptoms reported through a web application. Am J Clin Oncol. 2017;40(5):464–469. doi: 10.1097/COC.0000000000000189.
    1. van der Meij E, Anema JR, Leclercq WKG, Bongers MY, Consten ECJ, Schraffordt Koops SE, et al. Personalised perioperative care by e-health after intermediate-grade abdominal surgery: a multicentre, single-blind, randomised, placebo-controlled trial. Lancet. 2018;392(10141):51–59. doi: 10.1016/S0140-6736(18)31113-9.
    1. Montane E, Ibanez L, Vidal X, Ballarin E, Puig R, Garcia N, et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica. 2008;93(4):518–523. doi: 10.3324/haematol.12020.
    1. Vaht K, Goransson M, Carlson K, Isaksson C, Lenhoff S, Sandstedt A, et al. Incidence and outcome of acquired aplastic anemia: real-world data from patients diagnosed in Sweden from 2000–2011. Haematologica. 2017;102(10):1683–1690. doi: 10.3324/haematol.2017.169862.
    1. 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):543–553. doi: 10.1002/cam4.1953.
    1. Hillmen P, Roman E, Hill QA, Cullen MJ, Richards SJ, Smith A, et al. The Incidence and prevalence of paroxysmal nocturnal hemoglobinuria (PNH) and Survival of patients in Yorkshire. Blood. 2006;108(11):985. doi: 10.1182/blood.V108.11.985.985.
    1. Devos T, Meers S, Boeckx N, Gothot A, Deeren D, Chatelain B, et al. Diagnosis and management of PNH: review and recommendations from a Belgian expert panel. Eur J Haematol. 2018;101(6):737–749. doi: 10.1111/ejh.13166.
    1. Valdez JM, Scheinberg P, Nunez O, Wu CO, Young NS, Walsh TJ. Decreased infection-related mortality and improved survival in severe aplastic anemia in the past two decades. Clin Infect Dis. 2011;52(6):726–735. doi: 10.1093/cid/ciq245.
    1. Hochsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H. Supportive care in severe and very severe aplastic anemia. Bone Marrow Transplant. 2013;48(2):168–173. doi: 10.1038/bmt.2012.220.
    1. Schrezenmeier H, Muus P, Socie G, Szer J, Urbano-Ispizua A, Maciejewski JP, et al. Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry. Haematologica. 2014;99(5):922–929. doi: 10.3324/haematol.2013.093161.
    1. Patriquin CJ, Kiss T, Caplan S, Chin-Yee I, Grewal K, Grossman J, et al. How we treat paroxysmal nocturnal hemoglobinuria: a consensus statement of the Canadian PNH Network and review of the national registry. Eur J Haematol. 2019;102(1):36–52. doi: 10.1111/ejh.13176.
    1. Brodsky RA, Peffault de Latour R, Rottinghaus ST, Roth A, Risitano AM, Weitz IC, et al (2020) Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria. Haematologica. 10.3324/haematol.2019.236877
    1. Iivanainen S, Alanko T, Peltola K, Konkola T, Ekstrom J, Virtanen H, et al. ePROs in the follow-up of cancer patients treated with immune checkpoint inhibitors: a retrospective study. J Cancer Res Clin Oncol. 2019;145(3):765–774. doi: 10.1007/s00432-018-02835-6.
    1. Peltola MK, Lehikoinen JS, Sippola LT, Saarilahti K, Makitie AA. A novel digital patient-reported outcome platform for head and neck oncology patients-a pilot study. Clin Med Insights Ear Nose Throat. 2016;9:1–6. doi: 10.4137/CMENT.S40219.
    1. Aiyegbusi OL. Key methodological considerations for usability testing of electronic patient-reported outcome (ePRO) systems. Qual Life Res. 2020;29(2):325–333. doi: 10.1007/s11136-019-02329-z.
    1. Antunes B, Harding R, Higginson IJ, Euroimpact. Implementing patient-reported outcome measures in palliative care clinical practice: a systematic review of facilitators and barriers. Palliat Med. 2014;28(2):158–75. doi: 10.1177/0269216313491619.
    1. Weisshaar K, Ewald H, Halter J, Gerull S, Schonfeld S, Senft Y, et al. Development of a patient-reported outcome questionnaire for aplastic anemia and paroxysmal nocturnal hemoglobinuria (PRO-AA/PNH) Orphanet J Rare Dis. 2020;15(1):249. doi: 10.1186/s13023-020-01532-3.
    1. Mendoza TR, Dueck AC, Bennett AV, Mitchell SA, Reeve BB, Atkinson TM, et al. Evaluation of different recall periods for the US National Cancer Institute's PRO-CTCAE. Clin Trials. 2017;14(3):255–263. doi: 10.1177/1740774517698645.
    1. Jensen RE, Snyder CF, Abernethy AP, Basch E, Potosky AL, Roberts AC, et al. Review of electronic patient-reported outcomes systems used in cancer clinical care. J Oncol Pract. 2014;10(4):e215–e222. doi: 10.1200/JOP.2013.001067.
    1. Bruner DW, Bryan CJ, Aaronson N, Blackmore CC, Brundage M, Cella D, et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institute-sponsored clinical trials networks. J Clin Oncol. 2007;25(32):5051–5057. doi: 10.1200/JCO.2007.11.3324.
    1. Snyder CF, Aaronson NK, Choucair AK, Elliott TE, Greenhalgh J, Halyard MY, et al. Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations. Qual Life Res. 2012;21(8):1305–1314. doi: 10.1007/s11136-011-0054-x.
    1. Smith E, Lai JS, Cella D. Building a measure of fatigue: the functional assessment of Chronic Illness Therapy Fatigue Scale. PM R. 2010;2(5):359–363. doi: 10.1016/j.pmrj.2010.04.017.
    1. Zhang PI, Hsu CC, Kao Y, Chen CJ, Kuo YW, Hsu SL, et al. Real-time AI prediction for major adverse cardiac events in emergency department patients with chest pain. Scand J Trauma Resusc Emerg Med. 2020;28(1):93. doi: 10.1186/s13049-020-00786-x.
    1. Seo DW, Yi H, Park B, Kim YJ, Jung DH, Woo I, et al. (2020) Prediction of adverse events in stable non-variceal gastrointestinal bleeding using machine learning. J Clin Med. 9(8). 10.3390/jcm9082603
    1. Abu-El-Noor NI, Aljeesh YI, Bottcher B, Abu-El-Noor MK. Impact of a mobile phone app on adherence to treatment regimens among hypertensive patients: a randomised clinical trial study. Eur J Cardiovasc Nurs. 2020;0(0):1474515120938235. doi: 10.1177/1474515120938235.
    1. Greer JA, Jacobs JM, Pensak N, Nisotel LE, Fishbein JN, MacDonald JJ, et al. Randomized trial of a smartphone mobile app to improve symptoms and adherence to oral therapy for cancer. J Natl Compr Canc Netw. 2020;18(2):133–141. doi: 10.6004/jnccn.2019.7354.
    1. Frailley SA, Blakely LJ, Owens L, Roush A, Perry TS, Hellen V, et al. Electronic patient-reported outcomes (ePRO) platform engagement in cancer patients during COVID-19. J Clin Oncol. 2020;38(29):172. doi: 10.1200/JCO.2020.38.29_suppl.172.

Source: PubMed

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