National, clinical cohort study of late effects among survivors of acute lymphoblastic leukaemia: the ALL-STAR study protocol
Kjeld Schmiegelow,
Liv Andrés-Jensen,
Mette Tiedemann Skipper,
Kristian Mielke Christensen,
Pia Hedegaard Johnsen,
Katrine Aagaard Myhr,
Martin Kaj Fridh,
Kathrine Grell,
A. M. L. Pedersen,
Sune Leisgaard Mørck Rubak,
Martin Ballegaard,
Arne Hørlyck,
Rikke Beck Jensen,
Kim Gjerum Nielsen,
Ruta Tuckuviene,
Peder Skov Wehner,
Birgitte Klug Albertsen,
Thomas Leth Frandsen
Affiliations
Kjeld Schmiegelow
4 Department of Pediatrics and Adolescent Medicine, Juliane Marie Center, Rigshospitalet University Hospital, and Institute of Clinical Medicine, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark
Liv Andrés-Jensen
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Mette Tiedemann Skipper
Department of Pediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark
Kristian Mielke Christensen
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Pia Hedegaard Johnsen
Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark
Katrine Aagaard Myhr
Department of Cardiology, Copenhagen University Hospital, Copenhagen, Denmark
Martin Kaj Fridh
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Kathrine Grell
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
A. M. L. Pedersen
Section of Oral Medicine/Oral Biology and Immunopathology, Department of Odontology, University of Copenhagen, Copenhagen, Denmark
Sune Leisgaard Mørck Rubak
Department of Pediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark
Martin Ballegaard
Department of Neurology, Zealand University Hospital Roskilde, Roskilde, Denmark
Arne Hørlyck
Department of Radiology, Aarhus University Hospital, Aarhus, Denmark
Rikke Beck Jensen
Department of Growth and Reproduction, Copenhagen University Hospital, Copenhagen, Denmark
Kim Gjerum Nielsen
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Ruta Tuckuviene
Department of Pediatrics, Aalborg University Hospital, Aalborg, Denmark
Peder Skov Wehner
Department of Pediatric Hematology and Oncology, Odense University Hospital, Odense, Denmark
Birgitte Klug Albertsen
Department of Pediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark
Thomas Leth Frandsen
Department of Pediatrics and Adolescent Medicine, Copenhagen University Hospital, Copenhagen, Denmark
Introduction More than 90% of patients diagnosed with childhood acute lymphoblastic leukaemia (ALL) today will survive. However, half of the survivors are expected to experience therapy-related chronic or late occurring adverse effects, reducing quality of life. Insight into underlying risk trajectories is warranted. The aim of this study is to establish a Nordic, national childhood ALL survivor cohort, to be investigated for the total somatic and psychosocial treatment-related burden as well as associated risk factors, allowing subsequent linkage to nation-wide public health registers.Methods and analysis This population-based observational cohort study includes clinical follow-up of a retrospective childhood ALL survivor cohort (n=475), treated according to a common Nordic ALL protocol during 2008–2018 in Denmark. The study includes matched controls. Primary endpoints are the cumulative incidence and cumulative burden of 197 health conditions, assessed through self-report and proxy-report questionnaires, medical chart validation, and clinical examinations. Secondary endpoints include organ-specific outcome, including cardiovascular and pulmonary function, physical performance, neuropathy, metabolic disturbances, hepatic and pancreatic function, bone health, oral and dental health, kidney function, puberty and fertility, fatigue, and psychosocial outcome. Therapy exposure, acute toxicities, and host genome variants are explored as risk factors.Ethics and dissemination The study is approved by the Regional Ethics Committee for the Capital Region in Denmark (H-18035090/H-20006359) and by the Danish Data Protection Agency (VD-2018–519). Results will be published in peer-reviewed journals and are expected to guide interventions that will ameliorate the burden of therapy without compromising the chance of cure.