A thematic study: impact of COVID-19 pandemic on rare disease organisations and patients across ten jurisdictions in the Asia Pacific region

Wong

2021

Orphanet J Rare Dis

Self Cite

Background The threat and experience of pandemics occur differently for different groups. The rare disease population is at particular risk of being further marginalised during pandemics. In this study, our objective was to assess the hospital mortality patterns in the rare disease and the general populations during the coronavirus disease of 2019 (COVID-19) and severe acute respiratory syndrome (SARS) pandemics in Hong Kong. Methods All admission records during the COVID-19 pandemic (January 23–August 23, 2020) and SARS pandemic (March 11–June 30, 2003) were extracted from the local public healthcare database. Patients with rare diseases were identified using one or more of the 1084 10th version International Classification of Diseases and Related Health Problems (ICD-10) codes cross-referenced with 467 ORPHAcodes. Hospital mortality patterns were compared in patients with and without COVID-19/SARS infection. Admission records during the same period in 2019 and 2002 were retrieved for comparison. Results During the COVID-19 pandemic, 407,219 patients were admitted to one or more of the 43 public hospitals in Hong Kong, of which, 13,894 were patients with rare diseases (3.4%). A total of 4381 and 77 patients from the general and rare disease populations were infected with COVID-19. Rare disease patients had an adjusted 3.4 times odds of COVID-19-related hospital mortality compared with that of the general population (95% C.I. 1.24–9.41). COVID-19-related mortality was almost exclusively seen in patients ≥ 60 years. While age-related increase in mortality was also observed for the general population during the SARS pandemic, the pattern observed in the rare disease population was significantly different, with a 12.5 times higher SARS-related mortality observed in rare disease patients ≤ 18 years than those in the general population (12.5% vs 1.0%). Patients admitted during the same pandemic periods without coronavirus infection had a significantly higher hospital mortality compared with those admitted one year before the pandemic (p < 0.001). Conclusion This population-based study demonstrated the differential impacts of the COVID-19 and SARS pandemics on the rare disease population. In the era of budget and resource scarcity, this study warrants cautious healthcare planning, with consideration of the rare disease population in healthcare prioritisation.

Section: Discussionmentioning

confidence: 99%

Hospital mortality in patients with rare diseases during pandemics: lessons learnt from the COVID-19 and SARS pandemics

Wong

2021

Orphanet J Rare Dis

Self Cite

“…Generally, access to telemedicine was provided by 56% of all responding physicians. It is less than in other countries, where the percentage of centres with telehealth options was 90% [ 5 ], and about 70% of patients experienced this form of consultations [ 6 , 9 ]. Both healthcare providers and patients claim telemedicine is a useful strategy and should continue in certain cases (e.g.…”

Section: Discussionmentioning

confidence: 99%

“…Both healthcare providers and patients claim telemedicine is a useful strategy and should continue in certain cases (e.g. electronic prescriptions) after the pandemic [ 6 , 9 , 13 , 16 ].…”

Section: Discussionmentioning

confidence: 99%

“…Decreased healthcare availability and fear of SARS-CoV-2 infection caused a series of new challenges for people with rare metabolic diseases. Reports from organizations of rare disease patients worldwide [ 5 , 6 , 7 , 8 , 9 , 10 , 11 , 12 , 13 , 14 ], and rare disease healthcare providers [ 5 , 15 ] showed the scale of disturbances: from cancelled appointments, postponed i.v. treatments and hospitalisations to shortage of medical supply and impact on mental health.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

COVID-19 Pandemic and Patients with Rare Inherited Metabolic Disorders and Rare Autoinflammatory Diseases—Organizational Challenges from the Point of View of Healthcare Providers

Tobór-Świętek

Sykut-Cegielska²,

Bik-Multanowski

et al. 2021

JCM

COVID-19 pandemic is an organisational challenge for both healthcare providers and patients. People with rare inherited metabolic disorders (IMD) and rare autoinflammatory diseases (AD) are vulnerable patients whose well-being is deeply connected with regular follow-ups. This study aimed to assess how e one year of coronavirus pandemic has impacted the treatment of patients with IMD and AD in Poland. Surveys were distributed to all healthcare providers that coordinate the treatment of IMD and AD patients. Thirty-two responders (55%) answered the survey. They provide care to 1726 patients with IMD/AD, including 246 patients on dedicated treatment. In 35% of units, the regular appointments were disrupted, primarily because of patient infection. In 18 hospitals, remote visits were implemented, but only 66.6% of patients used this form of consultation. In 14/32 hospitals, administration of the therapy was delayed (median: 17.4 days). Forty-four patients suffered from SARS-COV-2 infection, in majority with mild symptoms. However, four adult patients developed complications, and one died following a SARS-COV-2 infection. Although most hospitals managed to maintain regular visits during the pandemic, more comprehensive implementation of telemedicine and switch to oral therapy or home infusions would be a reasonable solution for the current epidemic situation.

“…Failure to obtain timely treatment may predispose metabolic decompensations, affect developmental functioning, and even cause lifelong disability and death. 6 Multicenter studies including children with RIMDs during the COVID-19 pandemic have found that children and their families experienced difficulties in accessing health care, medications, and timely treatment, 10 - 12 however, have not examined all needs of these children like education, rehabilitation, and special services using the holistic framework proposed by the United Nations Convention on the Rights of the Child, stating that every child needs to live, survive, develop to optimal potential, and have education. 17…”

Section: Introductionmentioning

confidence: 99%

Unmet Needs of Children with Inherited Metabolic Disorders in the COVID-19 Pandemic

Akin¹,

Eminoğlu²,

Doğulu³

et al. 2022

Turk Arch Pediatrics

Objective Crucial information is lacking on unmet needs of children with rare inherited metabolic disorders during the coronavirus disease 2019 pandemic from low- and middle-income countries. We aimed to identify the unmet needs of children with rare inherited metabolic disorders from Turkey. Materials and Methods In a cross-sectional observational design, all children with rare inherited metabolic disorders aged 0-18 years followed at Ankara University School of Medicine Department of Pediatrics Pediatric Metabolism Division were recruited and interviewed via phone calls. The Expanded Guide for Monitoring Child Development enabled assessment of unmet needs and environmental context during coronavirus disease 2019 pandemic. Step-wise logistic regression analysis was used to determine independent factors associated with unmet needs. Results The sample comprised 229 children (54.1% boys) with rare inherited metabolic disorders (36.7% diet-dependent disorders). Most common diagnoses were amino acid metabolism disorders (40.2%). Of all, 29.3% of the mothers reported depression, 25.3% loss of job of family members during the pandemic. All children had unmet needs: at least 73.0% in health care, 96.8% in education, 78.3% in special services/rehabilitation. Having significant developmental delay and/or disability (odds ratio = 2.31, 95% CI: 1.14-4.67) emerged as the only independent factor associated with unmet needs in health care. Conclusion Children with rare inherited metabolic disorders and their families in Turkey experience unmet needs in many domains during coronavirus disease 2019 pandemic. Urgent action is needed to address the unmet needs of children with rare inherited metabolic disorders, especially those who has significant developmental delays and/or disabilities for this pandemic and possible future crisis.