VPRIV safely benefits Gaucher kids across all age groups, review finds
Approved ERT helps children with type 1 maintain stable blood levels
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Treatment with VPRIV (velaglucerase alfa) was generally safe and showed benefits — including stable blood levels — among children and adolescents with Gaucher disease type 1, a new review of published studies has found.
Evidence also suggests the approved enzyme replacement therapy (ERT) may help manage nonneurological symptoms in children with Gaucher type 3, a rarer form of the disease. Further studies are needed to clarify the therapy’s effects in this patient group, the researchers noted.
According to the scientists, these “findings suggest that [VPRIV] may be a beneficial option for [Gaucher type 1] across all paediatric age groups (0–18 years). Additionally, it might be considered a therapeutic option for non-neurological [Gaucher type 3] symptoms.”
The study, “Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups,” was published in the Orphanet Journal of Rare Diseases by researchers in Spain. It was funded by the Spanish branch of Takeda Pharmaceuticals, which markets VPRIV.
Gaucher disease is caused by mutations that impair the production or activity of glucocerebrosidase, an enzyme that’s needed to break down certain fatty molecules inside cells. Without enough of this enzyme, these molecules accumulate to toxic levels, leading to damage to organs such as the liver, spleen, and bones.
Type 1, the most common form of the disease — accounting for at least 90% of all cases — typically begins in childhood, though about one-third of patients don’t see symptoms until adulthood. Type 3 accounts for about 5% of Gaucher cases and is marked by neurological symptoms.
Trials of VPRIV did not include children 3 or younger
As an ERT, VPRIV delivers the missing glucocerebrosidase enzyme to patients via regular infusions into the bloodstream. This helps cells break down the excess fatty molecules, thereby easing disease symptoms.
The therapy is approved for the long-term treatment of children and adults with Gaucher type 1, which counts among its symptoms low levels of red blood cells. However, the pivotal clinical trials that led to its approval involved only patients ages 4 and older, which means there’s limited evidence about the treatment’s use in infants and toddlers.
VPRIV is also sometimes used in Gaucher disease type 3 to manage symptoms outside the nervous system, but its safety and effectiveness in pediatric patients with this disease type remain unclear.
To better understand the safety and effectiveness of VPRIV in younger patients, the research team reviewed data from 23 published studies reporting outcomes in children and adolescents treated with VPRIV.
Across these studies, 159 patients from 17 countries were enrolled, with ages ranging from 6 weeks to 17 years. Most patients (73%) had Gaucher disease type 1, while 26 (17%) had type 3. For 16 patients (10%), the disease type was not specified.
Slightly more than half of the children had never received treatment, while 57 had previously been treated with Cerezyme (imiglucerase), another approved ERT.
Therapy reduced liver, spleen size among newly treated patients
Overall, in the children with Gaucher type 1, VPRIV was generally well tolerated. Some patients experienced treatment-emergent side effects, most commonly headache, respiratory infections, and the common cold. Still, none of the children stopped treatment because of these side effects, and no deaths were reported.
The therapy’s use led to improvements across several disease measures. Blood parameters — including levels of hemoglobin, the oxygen-carrying protein in red blood cells — generally increased in previously untreated patients and remained stable in those who switched from Cerezyme. The same effect was seen for platelets, fragments of red blood cells that help in clotting.
Many untreated patients also experienced reductions in liver and spleen size after starting VPRIV, while organ volumes — which indicate the structural integrity and health status of an organ — usually remained stable in those who had previously been receiving treatment. Measures of bone marrow involvement also improved in untreated patients and tended to remain stable in previously treated ones.
Evidence of benefits in children with Gaucher type 3 was more limited. Available data suggested that VPRIV had a similar safety profile to that seen in children with type 1 disease, although a small number of patients experienced treatment-emergent side effects or developed antibodies against the therapy, which may reduce its effectiveness.
In these patients, treatment appeared to improve blood parameters and reduce liver and spleen size, particularly in those who had not previously received therapy. However, the benefits were mainly limited to symptoms outside the nervous system.
“The data recorded during our study suggest that [VPRIV] may be considered a safe treatment for patients with [Gaucher disease type 3],” the researchers wrote, adding that it may “improve [blood- and organ-related] symptoms in all ages of paediatric patients.”
This work is important because it describes the safety and efficacy of [VPRIV] in the treatment of paediatric patients with [Gaucher disease], given that early intervention influences the benefits accrued.
The team highlighted the need for further studies in this group, noting that “there is limited published evidence regarding the use of [VPRIV] in [Gaucher disease type 3].”
Overall, the findings suggest that VPRIV is a useful treatment option for pediatric patients with Gaucher disease type 1 across all age groups, according to the researchers. The therapy may also help manage nonneurological symptoms in Gaucher disease type 3, although evidence in this population remains limited, the team noted.
“This work is important because it describes the safety and efficacy of [VPRIV] in the treatment of paediatric patients with [Gaucher disease], given that early intervention influences the benefits accrued,” the scientists concluded.
