Publication details

Hematopoietic stem cell transplantation positively affects the natural history of cancer in Nijmegen breakage syndrome

Authors	WOLSKA-KUSNIERZ Beata PASTORCZAK Agata FENDLER Wojciech WAKULINSKA Anna DEMBOWSKA-BAGINSKA Bozena HEROPOLITANSKA-PLISZKA Edyta PIATOSA Barbara PIETRUCHA Barbara KALWAK Krzysztof USSOWICZ Marek PIECZONKA Anna DRABKO Katarzyna LEJMAN Monika KOLTAN Sylwia GOZDZIK Jolanta STYCZYNSKI Jan FEDOROVA Alina MIAKOVA Natalia DERIPAPA Elena KOSTYUCHENKO Larysa KŘENOVÁ Zdenka HLAVÁČKOVÁ Eva GENNERY Andrew SYKORA Karl-Walter GHOSH Sujal ALBERT Michael H BALASHOV Dmitry EAPEN Mary SVEC Peter SEIDEL Markus G KILIC Sara Sebnem TOMASZEWSKA Agnieszka WIESIK-SZEWCZYK Ewa KREINS Alexandra GREIL Johann BUECHNER Jochen LUND Bendik GREGOREK Hanna CHRZANOWSKA Krystyna MLYNARSKI Wojciech
Year of publication	2021
Type	Article in Periodical
Magazine / Source	Clinical Cancer Research
MU Faculty or unit	Faculty of Medicine
Citation
Web	https://clincancerres.aacrjournals.org/content/early/2020/10/20/1078-0432.CCR-20-2574.abstract
Doi	http://dx.doi.org/10.1158/1078-0432.CCR-20-2574
Keywords	Nijmegen breakage syndrome (NBS)
Description	Among 241 patients with NBS enrolled in the study from 11 countries, 151 (63.0%) patients were diagnosed with cancer. Incidence rates for primary and secondary cancer, tumor characteristics, and risk factors affecting overall survival (OS) were estimated. The cumulative cancer incidence was 40.21% ± 3.5% and 77.78% ± 3.4% at 10 years and 20 years of follow-up, respectively. Most of the tumors n = 95 (62.9%) were non-Hodgkin lymphomas. Overall, 20 (13.2%) secondary malignancies occurred at a median age of 18 (interquartile range, 13.7–21.5) years. The probability of 20 year overall survival (OS) for the whole cohort was 44.6% ± 4.5%. Patients who developed cancer had a shorter 20 year OS than those without malignancy (29.6% vs. 86.2%; P < ). A total of 49 patients with NBS underwent HSCT, including 14 patients transplanted before malignancy. Patients with NBS with diagnosed cancer who received HSCT had higher 20 year OS than those who did not (42.7% vs. 30.3%; P = 0.038, respectively). In the group of patients who underwent preemptive transplantation, only 1 patient developed cancer, which is 6.7 times lower as compared with nontransplanted patients [incidence rate ratio 0.149 (95% confidence interval, 0.138–0.162); P < 0.0001].