Table II. MMIHS and MSMDS: Summary of Clinical Data and Genotype of Individuals With Molecularly Confirmed Diagnosis.
| Phenotype | Megacystis-microcolon-intestinal hypoperistalsis syndrome (MMIHS) | Multisystemic smooth muscle dysfunction syndrome (MSMDS) | |
|---|---|---|---|
| Gene | ACTG2a | MYH11b | ACTA2c |
| Number of probands | 23 | 1 | 23 |
| Parental consanguinity | − | + (Algerian origin) | − |
| Inheritance | AD | AR | AD |
| Pathogenic variant | Heterozygous—all missense | Homozygous—stop codon | Heterozygous—all missense (codon 179) |
| De novo variant | 16/18d | No | 23/23 |
| Familial recurrence of the phenotype | 1/23e | − | − |
| Megacystis | 21/23 | + | 4/23 |
| Bladder dysfunction without | 2/23 | − | 10/23 |
| megacystis | |||
| Microcolon | 16/23 | + | − |
| Intestinal hypoperistalsis | 21/21f | + | 5/23 |
| Prune-belly | 2/20g | + | 2/23 |
| Mydriasis | − | − | 23/23 |
| Patent ductus arteriosus | − | − | 23/23 |
| Vascular involvement | 23/23 | ||
| Cerebrovascular | − | − | 21/23 |
| Aortic aneurism | − | − | 14/23 |
AD, autosomal dominant, AR, autosomal recessive, MMIHS, megacystis-microcolon-intestinal hypoperistalsis syndrome; MSMDS, multisystemicsmooth muscle dysfunction syndrome; +, feature present; —, feature absent. A summary of phenotype, genotype, and family history data of all individuals with ACTG2-related disorder, including MMIHS and milder phenotypes, is described in the supplementary material (SM5).
[Milewicz et al., 2010]; [Al-Mohaissen et al., 2012]; [Moller et al., 2012]; [Munot et al., 2012]; [Richer et al., 2012]; [Meuwissen et al., 2013]; [Moosa et al., 2013]; [Amans et al., 2014]; [Brodsky et al., 2014]; [Roulez et al., 2014]; [Yetman et al., 2015].
Unknown finding in five of the 23 individuals (18 evaluated patients), 16/18, one individual inherited the variant from the father, who presented with milder disease—family 34 [Wangler et al., 2014] and parental inheritance in a family due to probable gonadal mosacism—family 1 [Tuzovic et al., 2015].
The recurrence of MMIHS was reported in a family with two affected siblings and normal parents—family 1 [Tuzovic et al., 2015].
Unknown finding in two of the 23 individuals (21 evaluated patients).
Unknown finding in three of the 23 individuals (20 evaluated patients).