RT Journal Article
SR Electronic
T1 National, clinical cohort study of late effects among survivors of acute lymphoblastic leukaemia: the ALL-STAR study protocol
JF BMJ Open
JO BMJ Open
FD British Medical Journal Publishing Group
SP e045543
DO 10.1136/bmjopen-2020-045543
VO 11
IS 2
A1 Liv Andrés-Jensen
A1 Mette Tiedemann Skipper
A1 Kristian Mielke Christensen
A1 Pia Hedegaard Johnsen
A1 Katrine Aagaard Myhr
A1 Martin Kaj Fridh
A1 Kathrine Grell
A1 A. M. L. Pedersen
A1 Sune Leisgaard Mørck Rubak
A1 Martin Ballegaard
A1 Arne Hørlyck
A1 Rikke Beck Jensen
A1 Trine-Lise Lambine
A1 Kim Gjerum Nielsen
A1 Ruta Tuckuviene
A1 Peder Skov Wehner
A1 Birgitte Klug Albertsen
A1 Kjeld Schmiegelow
A1 Thomas Leth Frandsen
YR 2021
UL http://bmjopen.bmj.com/content/11/2/e045543.abstract
AB 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.