Health-Related Quality of Life and Health Resource Utilization in Patients with Primary Immunodeficiency Disease Prior to and Following 12 Months of Immunoglobulin G Treatment

John Routes, Beatriz Tavares Costa-Carvalho, Bodo Grimbacher, Kenneth Paris, Hans D Ochs, Alexandra Filipovich, Mary Hintermeyer, Karina Mescouto de Melo, Sarita Workman, Diane Ito, Xiaolan Ye, Patrick Bonnet, Josephine Li-McLeod, John Routes, Beatriz Tavares Costa-Carvalho, Bodo Grimbacher, Kenneth Paris, Hans D Ochs, Alexandra Filipovich, Mary Hintermeyer, Karina Mescouto de Melo, Sarita Workman, Diane Ito, Xiaolan Ye, Patrick Bonnet, Josephine Li-McLeod

Abstract

Purpose: Health-related quality of life (HRQOL) has not been examined in patients with predominant antibody deficiency both pre- and post-immunoglobulin G (IgG) treatment initiation. HRQOL and health resource utilization (HRU) were assessed in newly diagnosed patients with primary immunodeficiency disease (PIDD) pre- and 12 months post-IgG treatment initiation.

Methods: Adults (age ≥18 years) completed the 36-item Short Form Health Survey, version 2; pediatric patients (PP)/caregivers completed the Pediatric Quality of Life Inventory (PedsQL). Scores were compared with normative data from the US general population (GP) and patients with other chronic conditions (OCC).

Results: Seventeen adult patients (APs), 8 PPs, and 8 caregivers completed baseline assessments. APs had significantly lower baseline mean physical component summary scores versus GP (37.4 vs 50.5, p < 0.01) adults with chronic back pain (44.1, p < 0.05) or cancer (44.4, p < 0.05) and lower mental component summary scores versus GP (41.6 vs 49.2, p < 0.05). PPs had lower PedsQL total (63.1 vs 82.7), physical summary (64.5 vs 84.5), and psychosocial summary (62.5 vs 81.7) scores versus GP. Post-IgG treatment, 14 APs, 6 PPs, and 8 caregivers completed assessments. Hospital admissions (0.2 versus 1.8, p < 0.01), serious infections (3.3 versus 10.9, p < 0.01) and antibiotic prescriptions (3.0 versus 7.1; p < 0.01) decreased significantly overall. While APs reported significant improvement in role-physical (p = 0.01), general health (p < 0.01), and social functioning (p = 0.02) and caregivers in vitality (p < 0.01), PPs did not.

Conclusions: Pre-IgG treatment, patients with PIDD experienced diminished HRQOL versus GP and patients with OCC; post-treatment, HRU decreased and certain HRQOL aspects improved for APs and caregivers.

Keywords: Primary immunodeficiency; antibody deficiency; health resource utilization, short form health survey (SF-36); health-related quality of life; pediatric quality of life inventory (PedsQL).

Figures

Fig. 1
Fig. 1
SF-36 physical (a) and mental (b) summary and domain scoresa for adult patients with PIDD compared with patients with other chronic conditions and US general population. aPhysical functioning, role physical, bodily pain, and general health are domains of the physical component scores; vitality, social functioning, role emotional, and mental health are domains of the mental component score. *p < 0.05 compared with PIDD sample. **p < 0.01 compared with PIDD sample. CHF, congestive heart failure; COPD, chronic obstructive pulmonary disease; IgG, immunoglobulin G; MID, minimally important difference; PIDD, primary immunodeficiency diseases; SF-36, short form health survey; US, United States
Fig. 2
Fig. 2
SF-36 physical (a) and mental (b) component and domain scoresa of adult patients with PIDD at baseline and at 12 months following IgG treatment initiation. aPhysical functioning, role physical, bodily pain, and general health are domains of the physical component scores; vitality, social functioning, role emotional, and mental health are domains of the mental component score. ------- SF-36 US general population norms, Δ, difference. MID, minimally important difference; ns, not significant; PIDD, primary immunodeficiency disease; SF-36, 36-item Short Form Health Survey; US, United States
Fig. 3
Fig. 3
PedsQL total, physical, and psychosocial summary scores of pediatric patients with PIDD compared with pediatric patients with other chronic conditions and the US healthy population. The black bars represent the 95 % confidence intervals. PedsQL, pediatric quality of life; PIDD, primary immunodeficiency disease; US, United States
Fig. 4
Fig. 4
PedsQL scores among pediatric patients with PIDD at baseline and at 12 months following IgG treatment initiation. aMIDs were not available for summary and component scores. IgG, immunoglobulin G; MID, minimally important difference; ns, not significant; PedsQL, Pediatric Quality of Life; PIDD, primary immunodeficiency diseases; US, United States
Fig. 5
Fig. 5
PedsQL scores among individual pediatric patients with PIDD at baseline and at 12 months following IgG treatment initiation. IgG, immunoglobulin G; PedsQL, Pediatric Quality of Life; PIDD, primary immunodeficiency diseases
Fig. 6
Fig. 6
SF-36 physical (a) and mental (b) component and domain scoresa for caregivers of pediatric patients with PIDD at baseline and at 12 months following IgG treatment initiation. aPhysical functioning, role physical, bodily pain, and general health are domains of the physical component scores; vitality, social functioning, role emotional, and mental health are domains of the mental component score. ------- SF-36 US general population norms. Δ, difference. MID, minimally important difference; ns, not significant; PIDD, primary immunodeficiency disease; SF-36, 36-item Short Form Health Survey; US, United States

References

    1. Picard C, Al-Herz W, Bousfiha A, Casanova JL, Chatila T, Conley ME, et al. Primary immunodeficiency diseases: an update on the classification from the international union of immunological societies expert committee for primary immunodeficiency 2015. J Clin Immunol. 2015;35(8):696–726.
    1. Wood P, Stanworth S, Burton J, Jones A, Peckham DG, Green T, et al. Recognition, clinical diagnosis and management of patients with primary antibody deficiencies: a systematic review. Clin Exp Immunol. 2007;149:410–423. doi: 10.1111/j.1365-2249.2007.03432.x.
    1. Parvaneh N, Casanova JL, Notarangelo LD, Conley ME. Primary immunodeficiencies: a rapidly evolving story. J Allergy Clin Immunol. 2013;131:314–323. doi: 10.1016/j.jaci.2012.11.051.
    1. Cooper MA, Pommering TL, Korányi K. Primary immunodeficiencies. Am Fam Physician. 2003;68:2001–2008.
    1. Ballow M. Primary immunodeficiency disorders: antibody deficiency. J Allergy Clin Immunol. 2002;109:581–591. doi: 10.1067/mai.2002.122466.
    1. Chen Y, Stirling RG, Paul E, Hore-Lacy F, Thompson BR, Douglass JA. Longitudinal decline in lung function in patients with primary immunoglobulin deficiencies. J Allergy Clin Immunol. 2011;127:1414–1417. doi: 10.1016/j.jaci.2011.03.041.
    1. Rich AL, Le Jeune IR, McDermott L, Kinnear WJ. Serial lung function tests in primary immune deficiency. Clin Exp Immunol. 2008;151:110–113. doi: 10.1111/j.1365-2249.2007.03550.x.
    1. Boujaoude Z, Arya R, Rafferty W, Dammert P. Organising pneumonia in common variable immunodeficiency. BMJ Case Rep. 2013
    1. Skull S, Kemp A. Treatment of hypogammaglobulinaemia with intravenous immunoglobulin, 1973–93. Arch Dis Child. 1996;74:527–530. doi: 10.1136/adc.74.6.527.
    1. Ochs HD, Gupta S, Kiessling P, Nicolay U, Berger M. Safety and efficacy of self-administered subcutaneous immunoglobulin in patients with primary immunodeficiency diseases. J Clin Immunol. 2006;26:265–273. doi: 10.1007/s10875-006-9021-7.
    1. Gardulf A, Nicolay U, Asensio O, Bernatowska E, Böck A, Carvalho BC, et al. Rapid subcutaneous IgG replacement therapy is effective and safe in children and adults with primary immunodeficiencies—a prospective, multi-national study. J Clin Immunol. 2006;26:177–185. doi: 10.1007/s10875-006-9002-x.
    1. Chapel HM, Spickett GP, Ericson D, Engl W, Eibl M, Bjorkander J. The comparison of the efficacy and safety of intravenous versus subcutaneous immunoglobulin replacement therapy. J Clin Immunol. 2000;20:94–100. doi: 10.1023/A:1006678312925.
    1. Buckley RH, Schiff RI. The use of intravenous immune globulin in immunodeficiency disease. N Engl J Med. 1991;325:110–117. doi: 10.1056/NEJM199107113250207.
    1. Wasserman RL, Irani AM, Tracy J, Tsoukas C, Stark D, Levy R, et al. Pharmacokinetics and safety of subcutaneous immune globulin (human), 10 % caprylate/chromatography purified in patients with primary immunodeficiency disease. Clin Exp Immunol. 2010;161:518–526. doi: 10.1111/j.1365-2249.2010.04195.x.
    1. Wasserman RL, Melamed I, Kobrynski L, Strausbaugh SD, Stein MR, Sharkhawy M, et al. Efficacy, safety, and pharmacokinetics of a 10 % liquid immune globulin preparation (GAMMAGARD LIQUID, 10 %) administered subcutaneously in subjects with primary immunodeficiency disease. J Clin Immunol. 2011;31:323–331. doi: 10.1007/s10875-011-9512-z.
    1. Wasserman RL, Melamed I, Stein MR, et al. Recombinant human hyaluronidase-facilitated subcutaneous infusion of human immunoglobulins for primary immunodeficiency. J Allergy Clin Immunol. 2012;130(4):951–7. doi: 10.1016/j.jaci.2012.06.021.
    1. Immune Deficiency Foundation. Primary immune deficiency diseases in America: the third national survey of patients. 2007. . Accessed 29 May 2014.
    1. Quinti I, Di Pietro C, Martini H, Pesce AM, Lombardi F, Baumghartner M, et al. Health related quality of life in common variable immunodeficiency. Yonsei Med J. 2012;53:603–610. doi: 10.3349/ymj.2012.53.3.603.
    1. Chan A, Scalchunes C, Boyle M, Puck JM. Early vs. delayed diagnosis of severe combined immunodeficiency: a family perspective survey. Clin Immunol. 2011;138:3–8. doi: 10.1016/j.clim.2010.09.010.
    1. Buckley RH. Immune deficiency foundation diagnostic and clinical care guidelines for primary immunodeficiency diseases, 2nd ed. 2009. . Accessed 29 May 2014.
    1. Nicolay U, Kiessling P, Berger M, Gupta S, Yel L, Roifman CM, et al. Health-related quality of life and treatment satisfaction in North American patients with primary immunodeficiency diseases receiving subcutaneous IgG self-infusions at home. J Clin Immunol. 2006;26:65–72. doi: 10.1007/s10875-006-8905-x.
    1. Gardulf A, Borte M, Ochs HD, Nicolay U, Vivaglobin Clinical Study Group Prognostic factors for health-related quality of life in adults and children with primary antibody deficiencies receiving SCIG home therapy. Clin Immunol. 2008;126:81–88. doi: 10.1016/j.clim.2007.06.009.
    1. Winkelstein JA, Conley ME, James C, Howard V, Boyle J. Adults with X-linked agammaglobulinemia: impact of disease on daily lives, quality of life, educational and socioeconomic status, knowledge of inheritance, and reproductive attitudes. Medicine (Baltimore) 2008;87:253–258. doi: 10.1097/MD.0b013e318187ed81.
    1. Daly PB, Evans JH, Kobayashi RH, Kobayashi AL, et al. Home-based immunoglobulin infusion therapy: quality of life and patient health perceptions. Ann Allerty. 1991;67(5):504–510.
    1. Ramírez-Vargas N, Arablin-Oropeza SE, Mojica-Martínez D, Yamazaki-Nakashimada MA, de la Luz G-CM, Terán-Juárez LM, et al. Clinical and immunological features of common variable immunodeficiency in Mexican patients. Allergol Immunopathol (Madr) 2014;42:235–240. doi: 10.1016/j.aller.2013.01.007.
    1. Guaní-Guerra E, García-Ramírez UN, Jiménez-Romero AI, Velázquez-Ávalos JM, Gallardo-Martínez G, Mendoza-Espinoza FJ. Primary immunodeficiency diseases at reference and high-specialty hospitals in the state of Guanajuato, Mexico. Biomed Res Int. 2013;2013:187254. doi: 10.1155/2013/187254.
    1. Seymour B, Miles J, Haeney M. Primary antibody deficiency and diagnostic delay. J Clin Pathol. 2005;58:546–547. doi: 10.1136/jcp.2004.016204.
    1. Jörgensen GH, Gardulf A, Sigurdsson MI, Arnlaugsson S, et al. Health-related quality of life (HRQL) in immunodeficient adults with selective IgA deficiency compared with age- and gender-matched controls and identification of risk factors for poor HRQL. Qual Life Res. 2014;23(2):645–58. doi: 10.1007/s11136-013-0491-9.
    1. Kuburovic NB, Pasic S, Susic G, Stevanovic D, et al. Health-related quality of life, anxiety, and depressive symptoms in children with primary immunodeficiencies. Patient Prefer Adherence. 2014;8:323–30. doi: 10.2147/PPA.S58040.
    1. Aghamohammadi A, Montazeri A, Abolhassani H, Saroukhani S, et al. Health-related quality of life in primary antibody deficiency. Iran J Allergy Asthma Immunol. 2011;10(1):47–51.
    1. Zebracki K, Palermo TM, Hostoffer R, Duff K, et al. Health-related quality of life of children with primary immunodeficiency disease: a comparison study. Ann Allergy Asthma Immunol. 2004;93(6):557–61. doi: 10.1016/S1081-1206(10)61263-X.
    1. Soresina A, Nacinovich R, Bomba M, et al. The quality of life of children and adolescents with X-linked agammaglobulinemia. J Clin Immunol. 2009;29:501–507. doi: 10.1007/s10875-008-9270-8.
    1. Ware JE, Jr, Kosinski M, Gandek B. SF-36 health survey: manual and interpretation guide. Lincoln, RI: QualityMetric; 2000.
    1. Ware JE, Jr, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30:473–483. doi: 10.1097/00005650-199206000-00002.
    1. McHorney CA, Ware JE, Jr, Raczek AE. The MOS 36-item short-form health survey (SF-36): II. Psychometric and clinical tests of validity in measuring physical and mental health constructs. Med Care. 1993;31:247–263. doi: 10.1097/00005650-199303000-00006.
    1. Scoggins JF, Patrick DL. The use of patient-reported outcomes instruments in registered clinical trials: evidence from . Contemp Clin Trials. 2009;30:289–292. doi: 10.1016/j.cct.2009.02.005.
    1. Varni JW, Limbers CA, Burwinkle TM. Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 2007;5:43. doi: 10.1186/1477-7525-5-43.
    1. Varni JW, Burwinkle TM, Seid M. The PedsQL as a pediatric patient-reported outcome: reliability and validity of the PedsQL Measurement Model in 25,000 children. Expert Rev Pharmacoecon Outcomes Res. 2005;5:705–719. doi: 10.1586/14737167.5.6.705.
    1. Ware JE, Jr, Gandek B, Kosinski M, Aaronson NK, Apolone G, Brazier J, et al. The equivalence of SF-36 summary health scores estimated using standard and country-specific algorithms in 10 countries: results from the IQOLA Project. International Quality of Life Assessment. J Clin Epidemiol. 1998;51:1167–1170. doi: 10.1016/S0895-4356(98)00108-5.
    1. Maruish, ME (Ed.). User’s manual for the SF-36v2 health survey 3rd ed. Lincoln, RI: Quality Metric Incorporated.
    1. Kobrynski L, Powell RW, Bowen S. Prevalence and morbidity of primary immunodeficiency diseases, United States 2001–2007. J Clin Immunol. 2014;34(8):954–61. doi: 10.1007/s10875-014-0102-8.

Source: PubMed

Sponzoři a spolupracovníci

Zdravotní podmínky

Drogové intervence

3
Předplatit