Study Evaluates rhGH For Children with ISS and GHD

According to a recent article, the results of a new study show that using recombinant human growth hormone to treat children with idiopathic short stature and growth hormone deficiency aids in improving the patients’ height.

Idiopathic Short Stature

Idiopathic short stature is a condition in which the height of the individual is more than 2 SD below the corresponding mean height for a given age, sex, and population, in whom no identifiable disorder is present. It can be subcategorized into familial and non-familial ISS, and according to pubertal delay. It should be differentiated from dysmorphic syndromes, skeletal dysplasias, short stature secondary to a small birth size (small for gestational age, SGA), and systemic and endocrine diseases. ISS is the diagnostic group that remains after excluding known conditions in short children.

Growth Hormone Deficiency

Growth hormone deficiency (GHD) is a rare disorder characterized by the inadequate secretion of growth hormone (GH) from the anterior pituitary gland, a small gland located at the base of the brain that is responsible for the production of several hormones. GHD can be present from birth (congenital), resulting from genetic mutations or from structural defects in the brain. It can also be acquired later in life as a result of trauma, infection, radiation therapy, or tumor growth within the brain. A third category has no known or diagnosable cause (idiopathic). Childhood-onset GHD may be all three: congenital, acquired, or idiopathic. It results in growth retardation, short stature, and maturation delays reflected by the delay of lengthening of the bones of the extremities that is inappropriate to the chronological age of the child.

Symptoms:

A child with GHD is usually of normal size at birth. A few children may become hypoglycemic (low blood sugar) during the newborn period. Males may have a small penis (micropenis). Later, children with GHD may present with delayed rates of development of facial bones, slow tooth eruption, delayed lengthening of long bones, fine hair, and poor nail growth. They may also demonstrate truncal obesity, a high pitched voice, and delayed closure of the sutures of the skull, causing delayed closure of the fontanelles.

The Study

The real-world study had a total of 79 pre-puberty patients diagnosed with idiopathic short stature (ISS) and 95 pre-puberty patients diagnosed with growth hormone deficiency (GHD). The patients were all ones who had visited the Chengdu Women’s and Children’s Central Hospital between January 2010 and September 2019.

The growth indexes were recorded before receiving treatment for both groups, and again subsequently after treatment to evaluate the effect the recombinant human growth hormone (rhGH) had on patients. The growth indexes that were evaluated were chronological age (CA), bone age (BA), height standard deviation score (HtSDS), insulin-like growth factor-1 (IGF-1), and body mass index.

Results

Initially, the two groups growth indexes were not very different. Even after 2 years of rhGH treatment, the differences between the ISS and GHD groups were not significantly different. However, the differences in HtSDS were drastically different after only six months. It was then discovered that the most critical factor pertaining to efficacy of the treatment for the ISS group was the age at which patients started treatment, and for the GHD group the most critical factors were age and IGF-1 SDS.

Researchers concluded that treating patients with ISS and GHD with recombinant human growth hormone does greatly improve the heights of the patients. Furthermore, they also concluded that there was not a substantial difference in the growth rate between ISS patients and GHD patients at relatively high doses. The factor that most commonly influenced the effectiveness of the treatment was the age that patients started being treated with rhGH.

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