The follow-up of Chinese patients in mut-type methylmalonic acidemia
identified through expanded newborn screening
Abstract
Background: Isolated methylmalonic acidemia (MMA), an autosomal
recessive disorder of propionate metabolism, is usually caused by
mutations in the methylmalonyl-CoA mutase gene (mut-type MMA). Because
no universal consensus was made on whether mut-type MMA should be
included in newborn screening (NBS), we aimed to compare the outcome of
this disorder detected by NBS with that detected clinically and
investigate the influence of NBS on the disease course.
Methods: In this study, 168 patients with mut-type MMA
diagnosed by NBS were compared to 210 patients diagnosed after disease
onset while NBS was not performed. Clinical data of these patients from
7 metabolic centers in China were analyzed retrospectively, including
initial manifestations, biochemical metabolites, the responsiveness of
vitamin B12 therapy, and gene variation, to explore different factors on
the long-term outcome. Results: Among patients diagnosed
through NBS, 77 patients (45.8%) remained asymptomatic and 87 patients
(53.4%) showed favorable neurocognitive outcomes. In contrast with
individuals diagnosed clinically, only 30 cases (16.2%) developed
healthily. In our comparison of patients whether detected by NBS, the
age at diagnosis, the incidence of disease onset, the responsiveness of
vitamin B12, age at the start of vitamin B12 treatment, levels of
biochemical features before and after treatment, and the long-term
prognosis were remarkably different ( P<0.01). The
presence of disease onset and the blood C3/C2 ratio were more associated
with poor outcomes of patients whether identified by NBS. Importantly,
after considering NBS, the odd ratio of disease onset for poor outcome
decreased and the unresponsiveness to vitamin B12 increased.
Conclusion: Through preventing major disease-related events and
allowing an earlier treatment initiation, NBS is beneficial for the
prognosis of infants with mut-type MMA.