Health related quality of life measure in systemic pediatric rheumatic diseases and its translation to different languages: an international collaboration

Background: Rheumatic diseases in children are associated with significant morbidity and poor health-related quality of life (HRQOL). There is no health-related quality of life (HRQOL) scale available specifically for children with less common rheumatic diseases. These diseases share several features with systemic lupus erythematosus (SLE) such as their chronic episodic nature, multi-systemic involvement, and the need for immunosuppressive medications. HRQOL scale developed for pediatric SLE will likely be applicable to children with systemic inflammatory diseases.Findings: We adapted Simple Measure of Impact of Lupus Erythematosus in Youngsters (SMILEY (c)) to Simple Measure of Impact of Illness in Youngsters (SMILY (c)-Illness) and had it reviewed by pediatric rheumatologists for its appropriateness and cultural suitability. We tested SMILY (c)-Illness in patients with inflammatory rheumatic diseases and then translated it into 28 languages. Nineteen children (79% female, n= 15) and 17 parents participated. the mean age was 12 +/- 4 years, with median disease duration of 21 months (1-172 months). We translated SMILY (c)-Illness into the following 28 languages: Danish, Dutch, French (France), English (UK), German (Germany), German (Austria), German (Switzerland), Hebrew, Italian, Portuguese (Brazil), Slovene, Spanish (USA and Puerto Rico), Spanish (Spain), Spanish (Argentina), Spanish (Mexico), Spanish (Venezuela), Turkish, Afrikaans, Arabic (Saudi Arabia), Arabic (Egypt), Czech, Greek, Hindi, Hungarian, Japanese, Romanian, Serbian and Xhosa.Conclusion: SMILY (c)-Illness is a brief, easy to administer and score HRQOL scale for children with systemic rheumatic diseases. It is suitable for use across different age groups and literacy levels. SMILY (c)-Illness with its available translations may be used as useful adjuncts to clinical practice and research.Rutgers State Univ, Robert Wood Johnson Med Sch, New Brunswick, NJ 08903 USARutgers State Univ, Child Hlth Inst New Jersey, New Brunswick, NJ 08901 USAHosp Special Surg, New York, NY 10021 USAUniv Michigan, Ann Arbor, MI 48109 USARed Cross War Mem Childrens Hosp, Cape Town, South AfricaAin Shams Univ, Pediat Allergy Immunol & Rheumatol Unit, Cairo, EgyptAin Shams Univ, Pediat Rheumatol Pediat Allergy Immunol & Rheum, Cairo, EgyptKing Faisal Specialist Hosp & Res Ctr, Riyadh 11211, Saudi ArabiaCharles Univ Prague, Prague, Czech RepublicGen Univ Hosp, Prague, Czech RepublicUniv Hosp Motol, Dept Pediat, Prague, Czech RepublicAarhus Univ, Hosp Skejby, Aarhus, DenmarkRigshosp, Juliane Marie Ctr, DK-2100 Copenhagen, DenmarkUniv Med Ctr, Dept Pediat Immunol, Utrecht, NetherlandsWilhelmina Childrens Hosp, Utrecht, NetherlandsGreat Ormond St Hosp Sick Children, Children NHS Fdn Trust, Renal Unit, London, EnglandLyon Univ, Hosp Civils Lyon, Rheumatol & Dermatol Dept, Lyon, FranceMed Univ Innsbruck, A-6020 Innsbruck, AustriaPrim Univ Doz, Bregenz, AustriaHamburg Ctr Pediat & Adolescence Rheumatol, Hamburg, GermanyAsklepios Clin Sankt, Augustin, GermanyUniv Zurich, Childrens Hosp, Zurich, SwitzerlandAristotle Univ Thessaloniki, Pediat Immunol & Rheumatol Referral Ctr, GR-54006 Thessaloniki, GreeceIsrael Meir Hosp, Kefar Sava, IsraelSanjay Gandhi Postgrad Inst Med Sci, Lucknow, Uttar Pradesh, IndiaSemmelweis Univ, H-1085 Budapest, HungaryAnna Meyer Hosp, Florence, ItalyUniv Siena, Res Ctr System Autoimmune & Autoinflammatory Dis, I-53100 Siena, ItalyUniv Florence, Florence, ItalyOsped Pediat Bambino Gesu, IRCCS, Pediat Rheumatol Unit, Rome, ItalyUniv Genoa Pediat II Reumatol, Ist G Gaslini EULAR, Ctr Excellence Rheumatol, Genoa, ItalyUniv Cattolica Sacro Cuore, Inst Pediat, Rome, ItalyUniv Padua, Dept Pediat, Pediat Rheumatol Unit, Padua, ItalyYokohama City Univ, Sch Med, Yokohama, Kanagawa 232, JapanUniv Estadual Paulista, UNESP, Botucatu, SP, BrazilUniversidade Federal de São Paulo, Dept Pediat, São Paulo, BrazilUniv Estadual Campinas, Dept Med, Campinas, SP, BrazilUniv Fed Rio de Janeiro, Dept Pediat, Rio de Janeiro, BrazilUniv Estado do, Adolescent Hlth Care Unit, Div Pediat Rheumatol, Rio de Janeiro, BrazilUniv São Paulo, Fac Med, Childrens Inst, Dept Pediat,Pediat Rheumatol Unit, São Paulo, BrazilChildrens Inst, Pediat Rheumatol Unit, São Paulo, BrazilClin Pediat I, Cluj Napoca, RomaniaInst Rheumatol, Belgrade, SerbiaUniv Childrens Hosp, Univ Med Ctr Ljubljana, Ljubljana, SloveniaHead Rheumatol Hosp Pedro Elizalde, Buenos Aires, DF, ArgentinaHosp Gen Mexico City, Mexico City, DF, MexicoHosp Infantil Mexico Fed Gomez, Mexico City, DF, MexicoHosp San Juan Dios, Barcelona, SpainHosp Univ Valle Hebron, Barcelona, SpainMt Sinai Med Ctr, New York, NY 10029 USAMt Sinai Med Ctr, Miami Beach, FL 33140 USAComplejo Hosp Univ Ruiz & Paez, Bolivar, VenezuelaHacettepe Univ, Dept Pediat, Ankara, TurkeyIstanbul Univ, Cerrahpasa Med Sch, Istanbul, TurkeyFMF Arthrit Vasculitis & Orphan Dis Res Ctr, Inst Hlth Sci, Ankara, TurkeyUniv Calgary, Dept Pediat, Alberta Childrens Hosp, Res Inst, Calgary, AB T2N 1N4, CanadaUniversidade Federal de São Paulo, Dept Pediat, São Paulo, BrazilWeb of Scienc

Comparing Presenting Clinical Features in 48 Children With Microscopic Polyangiitis to 183 Children Who Have Granulomatosis With Polyangiitis (Wegener's) : an ARChiVe Cohort Study

OBJECTIVE: To uniquely classify children with microscopic polyangiitis (MPA), to describe their demographic characteristics, presenting clinical features, and initial treatments in comparison to patients with granulomatosis with polyangiitis (Wegener's) (GPA). METHODS: The European Medicines Agency (EMA) classification algorithm was applied by computation to categorical data from patients recruited to the ARChiVe (A Registry for Childhood Vasculitis: e-entry) cohort, with the data censored to November 2015. The EMA algorithm was used to uniquely distinguish children with MPA from children with GPA, whose diagnoses had been classified according to both adult- and pediatric-specific criteria. Descriptive statistics were used for comparisons. RESULTS: In total, 231 of 440 patients (64% female) fulfilled the classification criteria for either MPA (n\u2009=\u200948) or GPA (n\u2009=\u2009183). The median time to diagnosis was 1.6 months in the MPA group and 2.1 months in the GPA group (ranging to 39 and 73 months, respectively). Patients with MPA were significantly younger than those with GPA (median age 11 years versus 14 years). Constitutional features were equally common between the groups. In patients with MPA compared to those with GPA, pulmonary manifestations were less frequent (44% versus 74%) and less severe (primarily, hemorrhage, requirement for supplemental oxygen, and pulmonary failure). Renal pathologic features were frequently found in both groups (75% of patients with MPA versus 83% of patients with GPA) but tended toward greater severity in those with MPA (primarily, nephrotic-range proteinuria, requirement for dialysis, and end-stage renal disease). Airway/eye involvement was absent among patients with MPA, because these GPA-defining features preclude a diagnosis of MPA within the EMA algorithm. Similar proportions of patients with MPA and those with GPA received combination therapy with corticosteroids plus cyclophosphamide (69% and 78%, respectively) or both drugs in combination with plasmapheresis (19% and 22%, respectively). Other treatments administered, ranging in decreasing frequency from 13% to 3%, were rituximab, methotrexate, azathioprine, and mycophenolate mofetil. CONCLUSION: Younger age at disease onset and, perhaps, both gastrointestinal manifestations and more severe kidney disease seem to characterize the clinical profile in children with MPA compared to those with GPA. Delay in diagnosis suggests that recognition of these systemic vasculitides is suboptimal. Compared with adults, initial treatment regimens in children were comparable, but the complete reversal of female-to-male disease prevalence ratios is a provocative finding

Anaphylaxis to anakinra in a pediatric patient with systemic juvenile idiopathic arthritis successfully treated with canakinumab: a case-based review

We present the case of a 2-year-old boy with a history of necrotizing enterocolitis (NEC) with ileostomy diagnosed with systemic juvenile idiopathic arthritis (sJIA) at 10 months of age controlled on anti-interleukin-1 (anti-IL-1) therapy (anakinra). At 17 months of age, ileostomy reversal and bowel re-anastomosis was scheduled with anakinra discontinued 3 days prior to the surgery and steroids initiated in its place. Ten days postoperatively, anakinra was re-started for signs of sJIA flare. Three months later, he developed persistent peripheral eosinophilia and subsequent anaphylactic reaction 6 months postoperatively. The patient safely tolerated an alternative anti-IL-1 agent (canakinumab). Anaphylaxis to anakinra has not been previously reported in the pediatric literature. This case highlights an important issue in a pediatric patient with sJIA: safety of an alternate anti-IL-1 agent, following development of allergy to one initial agent

Relationship between health-related quality of life, disease activity and disease damage in a prospective international multicenter cohort of childhood onset systemic lupus erythematosus patients

Previously, we described associations between health-related quality of life (HRQOL) and disease-related factors among childhood onset systemic lupus erythematosus (cSLE) patients. Here we determined the relationship between HRQOL, disease activity and damage in a large prospective international cohort of cSLE. We compared HRQOL, disease activity and disease damage across different continents and examined the relationship between children's and parents' assessments of HRQOL. Patients with cSLE and their parents completed HRQOL measures at enrollment and 654 follow-up visits. Physicians assessed disease activity and damage. The multinational cohort ( n\u2009=\u2009467) had relatively low disease activity and damage. Patient and parent HRQOL scores were significantly correlated. Asian and European patients had the highest HRQOL, while South and North American patients had lower HRQOL scores. Renal, CNS, skin and musculoskeletal systems exhibited the highest levels of damage. North and South American and Asian patients were more likely to have disease damage and activity scores above median values, compared with Europeans. Asians were more likely to use cyclophosphamide/rituximab. Female gender, high disease activity and damage, non-White ethnicity, and use of cyclophosphamide and/rituximab were related to lower HRQOL. HRQOL domain scores continue to emphasize that SLE has widespread impact on all aspects of children's and parents' lives

Health related quality of life measure in systemic pediatric rheumatic diseases and its translation to different languages: an international collaboration

Background: Rheumatic diseases in children are associated with significant morbidity and poor health-related quality of life (HRQOL). There is no health-related quality of life (HRQOL) scale available specifically for children with less common rheumatic diseases. These diseases share several features with systemic lupus erythematosus (SLE) such as their chronic episodic nature, multi-systemic involvement, and the need for immunosuppressive medications. HRQOL scale developed for pediatric SLE will likely be applicable to children with systemic inflammatory diseases

Health related quality of life measure in systemic pediatric rheumatic diseases and its translation to different languages: an international collaboration

BACKGROUND: Rheumatic diseases in children are associated with significant morbidity and poor health-related quality of life (HRQOL). There is no health-related quality of life (HRQOL) scale available specifically for children with less common rheumatic diseases. These diseases share several features with systemic lupus erythematosus (SLE) such as their chronic episodic nature, multi-systemic involvement, and the need for immunosuppressive medications. HRQOL scale developed for pediatric SLE will likely be applicable to children with systemic inflammatory diseases. FINDINGS: We adapted Simple Measure of Impact of Lupus Erythematosus in Youngsters (SMILEY©) to Simple Measure of Impact of Illness in Youngsters (SMILY©-Illness) and had it reviewed by pediatric rheumatologists for its appropriateness and cultural suitability. We tested SMILY©-Illness in patients with inflammatory rheumatic diseases and then translated it into 28 languages. Nineteen children (79% female, n=15) and 17 parents participated. The mean age was 12±4 years, with median disease duration of 21 months (1-172 months). We translated SMILY©-Illness into the following 28 languages: Danish, Dutch, French (France), English (UK), German (Germany), German (Austria), German (Switzerland), Hebrew, Italian, Portuguese (Brazil), Slovene, Spanish (USA and Puerto Rico), Spanish (Spain), Spanish (Argentina), Spanish (Mexico), Spanish (Venezuela), Turkish, Afrikaans, Arabic (Saudi Arabia), Arabic (Egypt), Czech, Greek, Hindi, Hungarian, Japanese, Romanian, Serbian and Xhosa. CONCLUSION: SMILY©-Illness is a brief, easy to administer and score HRQOL scale for children with systemic rheumatic diseases. It is suitable for use across different age groups and literacy levels. SMILY©-Illness with its available translations may be used as useful adjuncts to clinical practice and research