Evidence map›Paper›PMID 34188580›Full record

ArticleJournal of blood medicine2021

Efficacy, Safety and Pharmacokinetic Results of a Phase III, Open-Label, Multicenter Study with a Plasma-Derived Von Willebrand Factor (VWF)/Factor VIII (FVIII) Concentrate in Pediatric Patients <12 Years of Age with Hemophilia A (SWIFTLY-HA Study).

Claudia Djambas Khayat, Genadi Iosava, Irina Romashevskaya, Oleksandra Stasyshyn, Marta Julia Lopez, Maria Teresa Pompa, Tobias Rogosch, Wilfried Seifert

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of blood medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01229007 (A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A), which is not on this map. Cited by 2 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01229007 phase3completednot on this map

A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A

TypeinterventionalSponsorCSL BehringRan2010 to 2014Enrolled35ConditionsHemophilia AArmsBiostate
3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Claudia Djambas KhayatHospital Hôtel Dieu de France, Saint Joseph University, Beirut, Lebanon.
Genadi IosavaJoint Stock Hematology and Transfusiology Research Institute, Tbilisi, Georgia.
Irina RomashevskayaRepublican Research Centre of Radiation Medicine and Human Ecology, Gomel, Belarus.ORCID 0000-0002-4968-4461
Oleksandra StasyshynInstitute of Blood Pathology and Transfusion Medicine, Lviv, Ukraine.ORCID 0000-0002-8366-5926
Marta Julia LopezGuatemala City Hospital Roosevelt, Guatemala, Guatemala.
Maria Teresa PompaMonterrey Nuevo Leon OCA Hospital (MIRC), Monterrey Nuevo Leon, Mexico.
Tobias RogoschCSL Behring, Clinical Research and Development, Marburg, Germany.ORCID 0000-0002-1674-1423
Wilfried SeifertCSL Behring, Clinical Research and Development, Marburg, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPlasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII; VONCENTO

methodsThis study (NCT01229007) investigated the pharmacokinetics (PK), efficacy and safety of pdVWF/FVIII in 35 previously treated (minimum 20 exposure days [EDs]) pediatric patients (<12 years) with severe hemophilia A. PK was evaluated with a single 50 IU FVIII/kg dose of pdVWF/FVIII. Efficacy and safety analyses were performed during on-demand treatment (n=17) or prophylaxis (n=18) for up to 100 EDs with a maximum study duration of 12 months.

resultsPK profiles were similar for patients aged <6 years and those aged 6-12 years, and, as expected, the youngest patients had an increased clearance. On-demand patients reported 320 non-surgical bleeding (NSB) events and received a median number of 29.0 infusions (median dose 34.2 IU FVIII/kg). Hemostatic efficacy was assessed by the investigator as excellent/good in all cases (24%/76%). The 18 patients in the prophylaxis arm experienced 173 NSB events (97 NSBs [56%] in three patients). Five patients (28%) had no NSB events. Overall, patients received a median number of 92 infusions (median dose 30.6 IU FVIII/kg). The majority of bleeds (92%) were successfully controlled with only one infusion. Hemostatic efficacy was assessed by the investigator as excellent (86%) or good (14%). Inhibitors occurred in three patients of which two were transient (low titer) and one persisted (high titer). These three patients had known risk factors for inhibitor development.

conclusionThis study demonstrated comparable PK profiles for pediatric patients aged <6 years and aged 6-12 years, and an excellent efficacy and safety profile in this population. The adverse events reported were mostly mild to moderate with inhibitor rates within the expected incidence range.

Indexed as

factor VIIIhemophilia Ahemostatic efficacyon-demand therapyprophylaxisvon Willebrand factor

Identifiers

PMID34188580
PMCPMC8232873

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.