Definition
The term "dwarfism" indicates a particular medical condition, characterized by a defect in the somatic growth of the patients who suffer from it.
More specifically, we talk about dwarfism when, indicatively - after puberty - male patients do not exceed 130 cm in height and female patients do not exceed 125 cm.
The types of dwarfism can be divided into two large groups: harmonic dwarfism - in which body proportions and sexual and intellectual development are usually normal - and disharmonic dwarfism, in which, instead, body proportions are not maintained .
Examples of harmonic dwarfism are pituitary dwarfism and that of Laron (also known as Laron syndrome). Dwarfisms caused by hypothyroidism, achondroplasia and Turner syndrome are examples of disharmonic dwarfism.
Causes
The factors that lead to the onset of dwarfism can be of different types, including abnormalities or genetic alterations, endocrine dysfunctions and metabolic alterations.
Symptoms
Dwarf patients have a decidedly low stature compared to the norm. In the forms of disharmonic dwarfism, the body proportions are not maintained and, moreover, in most cases, there is also a reduced development both intellectual and sexual.
Furthermore, dwarfism could lead to the onset of muscular atrophy, absence or delay of puberty, micropenia, insomnia and depression.
Information on Dwarfism - Drugs to Treat Dwarfism is not intended to replace the direct relationship between health professional and patient. Always consult your doctor and / or specialist before taking Dwarfism - Drugs to Treat Dwarfism.
drugs
The possibility of carrying out treatment or not and the type of therapy to be undertaken in patients with dwarfism varies according to the underlying cause of its development.
For example - in the case where dwarfism is caused by hormonal deficiencies - one can intervene by giving patients the missing hormones, as in the case of pituitary dwarfism, in the case of Turner syndrome or in the case of achondroplasia.
In some cases of disharmonic dwarfism, moreover, the doctor could decide to intervene surgically to try to correct the anomalies and the bone malformations that characterize this particular form of dwarfism.
Somatotropin
Somatotropin (also known as somatropin, growth hormone or GH, from the English "Growth Hormone") can be used in the treatment of some forms of dwarfism, such as pituitary dwarfism, Turner syndrome and achondroplasia.
It should be emphasized, however, that in the case of pituitary dwarfism and in the case of Turner syndrome, the administration of growth hormone may favor the growth of patients suffering from this disease, but this does not occur in patients suffering from achondroplasia, despite the however, administration of GH has proved to be useful also in the latter form of dwarfism.
Somatotropin (NutropinAq ®, Omnitrope ®) is available in pharmaceutical formulations suitable for parenteral administration.
The usual starting dose administered in adults is 0.15-0.3 mg of somatotropin, to be taken daily by subcutaneous injection. Depending on the response of each patient to therapy, the doctor may decide to increase the dose of the drug administered, until the minimum effective dose is reached for each individual.
In children of both sexes with growth hormone deficiency, the usual somatotropin dose is 0.025-0.035 mg / kg body weight per day, to be administered subcutaneously.
In girls with Turner syndrome, on the other hand, the dose of somatotropin usually used is 0.05 mg / kg of body weight per day, to be administered by subcutaneous injection (for more detailed information on the treatment of this syndrome, we recommend see the dedicated article "Drugs for Curing Turner Syndrome").
The treatment with somatotropin can last for quite a long time and, sometimes, can continue for life.
In any case, the exact dosage of medicine must be established by the doctor on an individual basis for each patient. Therefore, it is essential to follow the indications provided by it, both in terms of the amount of drug to be taken and the duration of therapy.
mecasermin
Mecasermin is nothing but the insulin-like growth factor of type 1 (or IGF-1) that is normally produced by our body, but obtained synthetically in the laboratory. This hormone is closely related to the somatotropin with which it promotes and promotes growth in children and adolescents.
Mecasermin (Increlex ®) is used in children and adolescents aged 2 to 18 years to treat a particular type of dwarfism: Laron's syndrome. The latter is a genetic disease characterized by growth hormone receptor abnormalities and reduced IGF-1 levels. Growth hormone levels, on the other hand, are usually normal. For this reason, in the treatment of this form of dwarfism the administration of somatotropin is not useful, but that of mecasermin.
Mecasermin is available as a solution for injection that must be administered subcutaneously. Generally, the dose of drug used is 0.04-0.12 mg / kg of body weight, to be taken twice a day.
The drug should be given shortly after a meal or snack, as it can have hypoglycemic insulin-like effects, therefore, it can cause a decrease in blood sugar levels.
However, when using mecasermin - to avoid the occurrence of dangerous side effects - it is essential to strictly follow all the indications provided by the doctor.
