Hindawi Publishing Corporation
Case Reports in Genetics
Volume 2013, Article ID 143781, 4 pages
http://dx.doi.org/10.1155/2013/143781
Case Report
Isolated p.H62L Mutation in the CYP21A2 Gene in a Simple
Virilizing 21-Hydroxylase Deficient Patient
Melisa Taboas,
1,2
Cecilia Fernández,
1
Susana Belli,
3
Noemi Buzzalino,
1
Liliana Alba,
1
and Liliana Dain
1,2
1
Centro Nacional de Gen` etica M´ edica, ANLIS “Dr. Carlos G. Malbr´ an”, Buenos Aires, Argentina
2
Instituto de Biolog` ıa y Medicina Experimetal, CONICET, Buenos Aires, Argentina
3
Divisi` on Endocrinolog´ ıa, Hospital Durand, Buenos Aires, Argentina
Correspondence should be addressed to Liliana Dain; ldain@fmc.fcen.uba.ar
Received 13 May 2013; Accepted 15 June 2013
Academic Editors: M. Fenger, S. F. Grant, and G. Vogt
Copyright © 2013 Melisa Taboas et al. Tis is an open access article distributed under the Creative Commons Attribution License,
which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Congenital adrenal hyperplasia due to 21-hydroxylase defciency accounts for 90%–95% of cases. Tis autosomal recessive disorder
has a broad spectrum of clinical forms, ranging from severe or classical, which includes the salt-wasting and simple virilizing
forms, to the mild late onset or nonclassical form. Most of the disease-causing mutations described are likely to be the consequence
of nonhomologous recombination or gene conversion events between the active CYP21A2 gene and its homologous CYP21A1P
pseudogene. Nevertheless, an increasing number of naturally occurring mutations have been found. Te change p.H62L is one
of the most frequent rare mutations of the CYP21A2 gene. It was suggested that the p.H62L represents a mild mutation that may
be responsible for a more severe enzymatic impairment when presented with another mild mutation on the same allele. In this
report, a 20-year-old woman carrying an isolated p.H62L mutation in compound heterozygosity with c.283-13A/C>G mutation is
described. Although a mildly nonclassical phenotype was expected, clinical signs and hormonal profle of the patient are consistent
with a more severe simple virilizing form of 21-hydroxylase defciency. Te study of genotype-phenotype correlation in additional
patients would help in defning the role of p.H62L in disease manifestation.
1. Introduction
Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase
defciency (OMIM 201910) accounts for 90%–95% of CAH
cases [1, 2]. Tis autosomal recessive disorder, which is the
most frequent inborn error of metabolism, has a broad
spectrum of clinical forms, ranging from severe or classical,
which includes the salt-wasting (SW) and simple virilizing
(SV) forms, to the mild late onset or nonclassical form of
CAH (NCCAH) [1].
Te afected enzyme, P450C21, is encoded by the
CYP21A2 gene, located together with a 98% nucleotide
sequence identity CYP21A1P pseudogene, on chromosome
6p21.3. Due to the high degree of identity between this gene
and its pseudogene, most of the disease-causing mutations
described are likely to be the consequence of nonhomologous
recombination or gene conversion events [3, 4]. In addition,
more than 130 rare point mutations that arise independently
of the pseudogene and that were found specifc to a pop-
ulation or a single family, have been described to date (for
details visit http://www.hgmd.cf.ac.uk). Most of the patients
are compound heterozygotes and their phenotype depends
on the underlying combination of mutations they have [5].
Te change p.H62L is one of the most frequent rare muta-
tions of the CYP21A2 gene. Based on patients’ phenotypes and
functional studies, it has been proposed that the p.H62L allele
represents a mild mutation, which may be responsible for a
more severe phenotype only when associated with another
mild mutation on the same allele [6, 7]. Moreover, by in silico
analyses using as a template the crystallized bovine CYP21
that shares 79% sequence identity with the human CYP21A2
protein, Haider et al. (2013) associate this mutation to the
mild form of the disease [8].
Herein we describe a recently genotyped patient from
our cohort carrying an isolated p.H62L mutation in one
allele and the severe c.283-13A/C>G mutation in the other