Leveraging space innovations for cancer breakthroughs on Earth

Introduction: Leukodystrophies are a heterogeneous group of inherited neurologic disorders. These disorders are indeed progressive and debilitating conditions with limited treatment options and high mortality rates. There is a deficiency in available data concerning both the mortality rates and the most common causes of death in leukodystrophies. Methods: We investigated the mortality rates, mean age at death, and the most common causes of death in a retrospective cohort of 165 Iranian pediatric patients who were diagnosed with leukodystrophies. Results: Death was recorded in 64 of 165 patients (38.8%) with a mean follow-up of 4.7 ± 3.25 years. The mean age of living patients was 7.9 ± 4.8 years and the mean age at death was 5.2 ± 3.9 years. Mortality rate of the entire cohort was 18.1% (30/165), 24.2% (40/165), and 35.7% (59/165) at 3, 5, and 10 years’ follow-up, respectively. The mean age at death was 2.13 ± 0.68 years, 2.67 ± 1.14 years, and 4.33 ± 2.73 years, at 3-, 5-, and 10-year follow-up from first symptom onset, correspondingly. However, there was a significant difference in the mean age at death in years in hypomyelinating leukodystrophies compared with other leukodystrophies (2.19 ± 0.19 standard error [SE], confidence interval [CI] 1.81-2.56; and 6.65 ± 0.62 SE (CI 5.42-7.87); log rank P = .0001, analysis of variance P = .0001). The most common causes of death were cardiopulmonary problems (47%), seizures (11%), sepsis (9%), and miscellaneous (33%). Conclusions: We proposed that a significant majority of childhood leukodystrophy deaths occur within the first 5 years of life, with a notable concentration during the initial 3 years. Further, the results of this study suggest the potential for targeted strategies to address the specific causes of death in children with leukodystrophies.