Damoctocog alfa pegol shows safety and maintained bleed protection in children with severe haemophilia AStudy finds haemophilia drug safe and effective in children over six months

European journal of haematology Published April 2, 2026 Study authors: Ozelo Margareth C, Luciani Matteo, Glosli Heidi, Kavakli Kaan, Samji Nasrin, Makris Gregory C, Tueck… PubMed ↗ NCT01775618 ↗ DOI ↗ Editorial oversight: Dr. Amelia Tan, PhD · Internal Medicine & Chronic Disease

AI-generated summary of the cited source, checked by automated accuracy review. How we work

Key Takeaway

Consider preliminary safety data for damoctocog alfa pegol in children; confirm in controlled trials.

This was a phase 3, multicentre, open-label, single-arm study evaluating damoctocog alfa pegol in 35 previously treated children aged 7 to <12 years with severe haemophilia A over 6.0 months. The primary outcome was the incidence of adverse events of special interest (AESI) leading to discontinuation during the first 4 exposure days; no AEs resulted in study drug discontinuation (0/35). The probability of <5% of patients experiencing an AESI was 92.2%. Secondary outcomes indicated bleed protection with prophylaxis was maintained.

Regarding safety, 21/35 children (60%) reported ≥1 adverse event, all of which were mild or moderate. Study drug-related AEs occurred in 3/35 children (8.6%). No AEs led to discontinuation, and 32/35 children completed the 6-month study. One loss of efficacy event (2.9%), considered an AESI, led to a temporary treatment interruption.

Key limitations include the open-label, single-arm study design, which lacks a comparator group. This design means the reported associations should not be interpreted as evidence of causation, and generalizability beyond the studied population and duration is uncertain. The findings provide preliminary evidence of safety and maintained efficacy for this specific patient group, but controlled studies are needed to establish comparative effectiveness and long-term safety.

Researchers studied a drug called damoctocog alfa pegol in children with severe haemophilia A. The study included 35 children, aged 7 to under 12, who had been treated for haemophilia before. The main goal was to check the drug's safety over the first few doses and its ability to prevent bleeds over six months.

No child had to stop the drug because of a bad reaction. About 60% of children had at least one mild or moderate side effect, but none were severe. The drug continued to protect children from bleeds. One child had a temporary break in treatment because the drug seemed to stop working as well for a time.

It is important to be careful with these results. This was an 'open-label' study, meaning everyone knew which drug was being given, and there was no comparison group getting a different treatment. This makes it harder to be certain about the drug's effects. The study was also relatively small and only lasted six months.

For now, this study suggests the drug may be a safe option for children in this age group, but longer and more controlled studies are needed to confirm these early findings.

What this means for you:

Early study shows a haemophilia drug was safe in children, but more research is needed to be sure.

Study Details

Study typePhase3

EvidenceLevel 2

Follow-up6.0 mo

PublishedApr 2026

PMID41486550

TrialNCT01775618

View Original Abstract ↓

BACKGROUND: In the earlier PROTECT VIII Kids study (NCT01775618), damoctocog alfa pegol was efficacious for prevention and treatment of bleeds in children aged < 12 years with severe haemophilia A. OBJECTIVE: Assess the safety of damoctocog alfa pegol, including hypersensitivity and loss of efficacy (LoE) due to an immune response to polyethylene glycol, in children aged 7 to < 12 years with severe haemophilia A. METHODS: Alfa-PROTECT is a phase 3, multicentre, open-label, single-arm study (NCT05147662). Primary endpoint was the incidence of adverse events of special interest (AESI) leading to discontinuation during the first 4 exposure days. RESULTS: Overall, 35 children enrolled; 32 completed the 6-month study, 21 (60%) reported ≥ 1 AE. Median (range) treatment duration was 182 (172-198) days. All AEs were mild/moderate; 3/35 children (8.6%) had study drug-related AEs. One (2.9%) LoE event was considered an AESI, and led to temporary treatment interruption. No AEs resulted in study drug discontinuation. The probability of < 5% of patients experiencing an AESI was 92.2%. Bleed protection was maintained with damoctocog alfa pegol prophylaxis. CONCLUSIONS: These data confirm the safety profile of damoctocog alfa pegol in children aged 7 to < 12 years with severe haemophilia A. Secondary endpoints indicate treatment was efficacious. TRIAL REGISTRATION: The Alfa-PROTECT trial is registered at ClinicalTrial.gov (NCT05147662).

Damoctocog alfa pegol shows safety and maintained bleed protection in children with severe haemophilia AStudy finds haemophilia drug safe and effective in children over six months

Study Details

Biomarkers and clinical factors show statistical associations with sepsis-induced coagulopathy in 37,459 patients

Your Blood May Signal a Deadly Clotting Crisis Before It Strikes

Clinical research that matters. Delivered to your inbox.

Damoctocog alfa pegol shows safety and maintained bleed protection in children with severe haemophilia AStudy finds haemophilia drug safe and effective in children over six months

More on Hemophilia A

Study Details

Biomarkers and clinical factors show statistical associations with sepsis-induced coagulopathy in 37,459 patients

Your Blood May Signal a Deadly Clotting Crisis Before It Strikes

Clinical research that matters. Delivered to your inbox.

Related in Hematology

From Other Specialties