If your kid has growth hormone deficiency, you may have to decide whether to give him daily or weekly somatropin. These drugs work by increasing the amount of growth hormone in the body. This is important because they help with development and growth, and are especially helpful for children with physical disabilities. However, there are some drawbacks to both weekly and daily somatropin, so it’s important to know exactly what you are getting yourself into.
Weekly vs daily somatropin
There is debate on which type of somatropin is better: weekly or daily. These treatments are based on the same molecule, but the action is different in the body and thus is more convenient and safer. Once weekly somatropin has been shown to provide comparable results to daily injections of the same molecule. This is important because the molecule has a half-life of about 18 to 36 hours. However, a recent real-world study found that the proportion of children who discontinued therapy is quite large.
This was an observational study using a database of IQVIA Medical Research Data. It included prescriptions claimed in UK clinics for children with growth hormone deficiency. Children with more than one prescription for somatropin were included in the analysis. The top three reasons for switching from one to the other were related to the frequency of injections.
There were 224 participants in this randomized, double-blind, placebo-controlled trial in 21 countries. Each participant was given a test injection of somatropin in the morning. Growth measurements were obtained during baseline and follow-up periods. Common growth measures were used, including the height standard deviation score (SDS) and the HV, which was measured at months 6 and 12. At the end of the 12-month treatment period, the SDS was approximately similar to that at months six and twelve.
A separate cohort received weekly somatropin. Both groups had similar growth outcomes, although the change in height velocity was slightly higher in the once-weekly group. The fliGHt trial, which surveyed 99 children, measured the same thing.
To determine the best possible dose, the authors examined the clinical effects of two different somatropin preparations, which were compared for efficacy, safety, and convenience. For comparison, the Somatrogon(c) group was injected every other day, while the Genotropin group was injected weekly. In terms of safety, the most serious adverse event was hypoparathyroidism. Other common adverse events, like pharyngitis, were less frequent in the once-weekly group.
The best thing about once-weekly somatropin was that it was well tolerated by most patients, including adolescents. Although there was a small number of reported treatment-emergent adverse events, the overall incidence of treatment-related side effects was surprisingly low. Another factor that contributed to the success of the once-weekly regimen was the presence of a supportive care team that is familiar with the recommended monitoring and care for patients with GHD.
There were also several other good news stories from this study. For example, the study uncovered the existence of a non-inferior lonapegsomatropin product in the UK market, which has a similar safety profile to rhGH. Finally, once-weekly somatropin has a lower risk of discontinuation, which is especially important in adolescents. Despite these findings, there is still room for improvement and more studies are needed.
Efficacy and safety of weekly somatrogon vs daily somatropin in children with growth hormone deficiency
The efficacy and safety of once-weekly somatropin versus daily GENOTROPIN(r) (somatropin for injection) in children with growth hormone deficiency (GHD) were evaluated in a phase 3 global trial. Two hundred twenty-four pre-pubertal children with GHD were randomized to receive either once-weekly somatrogon or daily GENOTROPIN(r). All patients had blood samples collected at months 3, 9, and 12 to assess growth hormone and insulin-like growth factor-1 concentrations. Those who had elevated IGF-1 levels were given reduced doses of study treatment.
Mean annual height velocity was similar between the Genotropin and somatrogon groups. A higher percentage of subjects in the somatrogon group reported injection site pain. The majority of adverse events (AEs) were mild, with no deaths reported. Some serious AEs, such as influenza and hypoparathyroidism, were also reported. For the somatrogon group, the primary endpoint was height velocity at 12 months, while for the Genotropin group, it was a change from baseline in height SDS at month 24. However, there was an overall higher change in height velocity in the somatrogon group.
The trial included a global, open-label, multicenter long-term extension study. Children who had completed the Phase 3 trial could switch to the extension study. Ninety-five percent of the patients switched. This resulted in data collection from more than 2,600 children. Among them, 97 children were excluded due to other causes of short stature. During the trial, a total of 19 SAEs occurred, including traumatic fracture, febrile convulsion, hypoparathyroidism, and pharyngitis. These SAEs were reported by only 2 subjects in the Genotropin group.
The primary and secondary objectives of the study were to evaluate the safety and efficacy of once-weekly somatrogon compared to daily GENOTROPIN(r) in Japanese children with GHD. In addition, the trial evaluated other growth parameters and the PK profile of three different doses of once-weekly somatrogon.
Primary and secondary outcomes were assessed using Kaplan-Meier methods to assess persistence over time. The 12-month follow-up cohort had an overall persistence of 38.1% for 8-11-year-olds and 80.6% for 12- to 15-year-olds. For 8- to 11-year-olds, an average time to discontinuation was 2.9 months. Similarly, a 48-month follow-up cohort had an average of 38.1% persistence for 12- to 15-year-olds and 69.2% persistence for 3- to 7-year-olds.
The study used longitudinal, non-identified electronic health record (EHR) data from the IQVIA Medical Research Data database. The IQVIA database contained prescriptions for children > 3 years old, but only those required for patient care were obtained. Since the sample size was small, statistical power was limited. There were also substantial amounts of missing data that may have affected the results. Therefore, some of the findings may have been biased.
The proportion of subjects who reported lower injection site pain was significantly greater in the Genotropin group. Moreover, the overall incidence of injection site pain was comparable between the two groups. At months 4, 7, and 9 of the study, a total of 5 subjects in the somatrogon group had IGF-1 SDS values above +2 on consecutive assessments. Dose adjustments were made for all of these patients. Those with repeated elevated levels of IGF-1 were given reduced doses of study treatment.
Skytrofa
Skytrofa is a human growth hormone that has been approved by the United States Food and Drug Administration for children with growth hormone deficiency. This medicine is a type of somatropin prodrug, which is designed to be given by subcutaneous injection once weekly. It was developed by Ascendis Pharma, a pharmaceutical company. The medication is available through pharmacies in four-dose cartons, for once-weekly administration.
The medication is used in children who are between the ages of one and 26 pounds, or have a height of at least 25 pounds. Children in the United States, Japan, and Greater China are eligible for the drug. In addition, the medicine is being studied in adult patients with GHD.
SKYTROFA is the first sustained-release growth hormone product that can be administered weekly under the skin. Other FDA-approved hGH formulations for pediatric patients require daily use. While this may seem convenient, there are some potential risks to this form of treatment. Patients may develop neoplasms, intracranial hypertension, and Type 2 diabetes. For this reason, it is important to discuss the risks and benefits of using this therapy with the physician.
The drug’s most common side effects are fever, cough, and vomiting. Although these symptoms are generally mild, they can be serious if they become chronic. They can also lead to joint pain, muscle pain, and fluid retention. If these symptoms continue for more than a month, the patient should consult a doctor. Also, patients may develop a low thyroid hormone level, which can be aggravated by SKYTROFA. Additionally, patients with low thyroid function may need to take additional glucocorticoids during treatment.
Before treating a child with SKYTROFA, it is important to discuss possible adverse reactions. Patients should be informed of the most common side effects, as well as those that can occur more rarely. Some of the more serious side effects include scoliosis, kidney damage, heart disease, and thyroid problems. These complications can be particularly dangerous in kids who have preexisting scoliosis, and patients should have their condition monitored closely.
Among the other potential complications of SKYTROFA are hemorrhage and pancreatitis. However, these problems are not common, and they can be treated successfully. SKYTROFA also can interact with other medicines, such as glucocorticoids, insulin, or antihyperglycemic agents. Lastly, patients with type 2 diabetes may need to adjust their insulin or medications. Considering all these factors, it is important to discuss the risks and side effects with the physicians and parents before deciding to give Skytrofa to a child.
In addition to the aforementioned complications, patients who have a condition such as a slipped capital femoral epiphysis, or a closed epiphysis, should not be treated with SKYTROFA. There have been reports of severe injury and death in this category of patients.