Jacqueline Soraru1, Nicole Isbel2, Germaine Wong3,4,5, Patrick Toby Coates6,7, Murty Mantha8, Abu Abraham9, Rajiv Juneja10, Danny Hsu11, Fiona Brown12, Bhadran Bose13, David Mudge2, Robert Carroll6, Joshua Kausman14, Peter Hughes15, Thomas Barbour15, Anne Durkan16, Peter Mount17, Darren Lee18, Nicholas Larkins19,20, Dwarakanathan Ranganathan21, Wai H Lim1,20. 1. Department of Renal Medicine, Sir Charles Gairdner Hospital, Perth, Western Australia, Australia. 2. Department of Nephrology, Princess Alexandra Hospital, Brisbane, Queensland, Australia. 3. Centre for Transplant and Renal Research, Westmead Hospital, Sydney, New South Wales, Australia. 4. Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, New South Wales, Australia. 5. School of Public Health, University of Sydney, Sydney, New South Wales, Australia. 6. Central and Northern Renal and Transplantation Service, Royal Adelaide Hospital, Adelaide, South Australia, Australia. 7. Adelaide Health and Medical Sciences, University of Adelaide, Adelaide, South Australia, Australia. 8. Department of Nephrology, Cairns Base Hospital, Cairns, Queensland, Australia. 9. Department of Nephrology and Renal Transplant, Fiona Stanley Hospital, Perth, Western Australia, Australia. 10. Flinders Medical Centre, Adelaide, South Australia, Australia. 11. Department of Haematology, Liverpool Hospital, Sydney, New South Wales, Australia. 12. Department of Nephrology, Monash Medical Centre, Melbourne, Victoria, Australia. 13. Department of Nephrology, Nepean Hospital, Blue Mountains, New South Wales, Australia. 14. Department of Nephrology and Renal Transplantation, The Royal Children's Hospital, Melbourne, Victoria, Australia. 15. Department of Nephrology and Transplantation, The Royal Melbourne Hospital, Melbourne, Victoria, Australia. 16. Department of Nephrology, The Children's Hospital at Westmead, Sydney, New South Wales, Australia. 17. Department of Nephrology, Austin Health, Melbourne, Australia. 18. Department of Renal Medicine, Eastern Health Clinical School, Monash University Melbourne, Melbourne, Victoria, Australia. 19. Department of Nephrology and Hypertension, Perth Children's Hospital, Perth, Western Australia, Australia. 20. School of Medicine, University of Western Australia, Perth, Western Australia, Australia. 21. Kidney Health Service, Royal Brisbane and Women's Hospital, Brisbane, School of Medicine, Griffith University, Mount Gravatt, Queensland, Australia.
Abstract
AIMS: To describe the baseline characteristics and treatment of Australian patients diagnosed with atypical haemolytic uraemic syndrome (aHUS) reported to the Global aHUS Registry. METHODS: Descriptive analysis of the Australian cohort with aHUS (n = 106) was undertaken for demographics, disease characteristics and prior treatment with eculizumab; comparing with the global cohort (n = 1688) for certain pre-specified disease characteristics. RESULTS: In Australia, almost two-thirds of patients diagnosed with aHUS were female and over 80% of patients were Caucasians, with similar proportions reported in the global cohort. Less than 6% of patients in the Australia and global cohorts were reported to have a history of autoimmune disease (4% vs 2%, respectively; P = .21) or cancer (5% vs 5%, respectively; P = .93), conditions that have been associated with secondary HUS. In the Australian cohort, 26% had received a kidney transplant and 68% of patients had received eculizumab. Kidneys were the most common organ involvement, followed by gastrointestinal tract (26%) and cardiovascular system (19%), with 35% of patients reported to have had at least two organs involved within 6 months prior to baseline visit or entry into the registry. Complement factor H was the most common pathogenic complement gene variant in the Australian patients. CONCLUSION: Data from the aHUS registry confirms and defines region-specific disease characteristics among a selected group of Australian children and adults with aHUS reported to the registry. Ongoing and more inclusive data will provide further information about temporal trends and treatment outcomes, representing a unique opportunity for clinicians and researchers to further develop knowledge surrounding this rare disease.
AIMS: To describe the baseline characteristics and treatment of Australian patients diagnosed with atypical haemolytic uraemic syndrome (aHUS) reported to the Global aHUS Registry. METHODS: Descriptive analysis of the Australian cohort with aHUS (n = 106) was undertaken for demographics, disease characteristics and prior treatment with eculizumab; comparing with the global cohort (n = 1688) for certain pre-specified disease characteristics. RESULTS: In Australia, almost two-thirds of patients diagnosed with aHUS were female and over 80% of patients were Caucasians, with similar proportions reported in the global cohort. Less than 6% of patients in the Australia and global cohorts were reported to have a history of autoimmune disease (4% vs 2%, respectively; P = .21) or cancer (5% vs 5%, respectively; P = .93), conditions that have been associated with secondary HUS. In the Australian cohort, 26% had received a kidney transplant and 68% of patients had received eculizumab. Kidneys were the most common organ involvement, followed by gastrointestinal tract (26%) and cardiovascular system (19%), with 35% of patients reported to have had at least two organs involved within 6 months prior to baseline visit or entry into the registry. Complement factor H was the most common pathogenic complement gene variant in the Australian patients. CONCLUSION: Data from the aHUS registry confirms and defines region-specific disease characteristics among a selected group of Australian children and adults with aHUS reported to the registry. Ongoing and more inclusive data will provide further information about temporal trends and treatment outcomes, representing a unique opportunity for clinicians and researchers to further develop knowledge surrounding this rare disease.
Authors: Maria Helena Vaisbich; Luís Gustavo Modelli de Andrade; Precil Diego Miranda de Menezes Neves; Lílian Monteiro Pereira Palma; Maria Cristina Ribeiro de Castro; Cassiano Augusto Braga Silva; Maria Izabel Neves de Holanda Barbosa; Maria Goretti Moreira Guimarães Penido; Oreste Ângelo Ferra Neto; Roberta Mendes Lima Sobral; Silvana Maria Carvalho Miranda; Stanley de Almeida Araújo; Igor Gouveia Pietrobom; Henrique Mochida Takase; Cláudia Ribeiro; Rafael Marques da Silva; César Augusto Almeida de Carvalho; David José Barros Machado; Ana Mateus Simões Teixeira E Silva; Andreia Ribeiro da Silva; Enzo Ricardo Russo; Flávio Henrique Soares Barros; Jarinne Camilo Landim Nasserala; Luciana Schmitt Cardon de Oliveira; Lucimary de Castro Sylvestre; Rafael Weissheimer; Sueli Oliveira Nascimento; Gilson Bianchini; Fellype de Carvalho Barreto; Valéria Soares Pigozzi Veloso; Patrícia Marques Fortes; Vinicius Sardão Colares; Jaelson Guilhem Gomes; André Falcão Pedrosa Leite; Pablo Girardelli Mendonça Mesquita; Osvaldo Merege Vieira-Neto Journal: Clin Kidney J Date: 2022-04-11