Evidence map›Paper›PMID 36911027›Full record

ArticleFrontiers in pediatrics2023

Real pandemic world results of pharmacokinetic-tailored personalized prophylaxis of bleeds in Polish children and adolescents with severe hemophilia A.

Tomasz Urasiński, Klaudia Paczóska, Wanda Badowska, Halina Bobrowska, Łucja Dakowicz, Grzegorz Dobaczewski, Elżbieta Latos-Grażyńska, Grażyna Karolczyk, Anna Klukowska, Andrzej Kołtan and 13 more

Abstract read
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In one paragraph

Article in Frontiers in pediatrics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
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

23 authors.

Tomasz UrasińskiDepartment of Pediatrics, Hemato-Oncology and Gastroenterology, Pomeranian Medical University, Szczecin, Poland.
Klaudia PaczóskaDepartment of Pediatrics, Hemato-Oncology and Gastroenterology, Pomeranian Medical University, Szczecin, Poland.
Wanda BadowskaDepartment of Pediatric Oncology and Hematology, School of Medicine, University of Warmia Mazury, Olsztyn, Poland.
Halina BobrowskaWielkopolska Child Health Center, Poznań, Poland.
Łucja DakowiczDepartment of Pediatric Oncology and Hematology, Medical University of Białystok, Białystok, Poland.
Grzegorz DobaczewskiDepartment of Bone Marrow Transplantation, Pediatric Oncology and Hematology, Wrocław Medical University, Wrocław, Poland.
Elżbieta Latos-GrażyńskaDepartment of Bone Marrow Transplantation, Pediatric Oncology and Hematology, Wrocław Medical University, Wrocław, Poland.
Grażyna KarolczykII Department of Pediatrics, Collegium Medicum, Jan Kochanowski University, Kielce, Poland.
Anna KlukowskaDepartment of Pediatric Oncology, Hematology and Transplantation, Medical University of Warsaw, Warszawa, Poland.
Andrzej KołtanDepartment of Pediatrics, Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University, Bydgoszcz, Poland.
Magdalena WojdalskaDepartment of Pediatrics, Oncology and Hematology, Medical University of Lodz, Łódź, Poland.
Paweł ŁagunaDepartment of Pediatric Oncology, Hematology and Transplantation, Medical University of Warsaw, Warszawa, Poland.
Maciej NiedźwiedzkiDepartment of Pediatrics, Hematology and Oncology, Medical University of Gdańsk, Gdańsk, Poland.
Danuta PietrysDepartment of Pediatric Oncology and Hematology, Collegium Medicum, Jagiellonian University, Kraków, Poland.
Julia Radoń-ProskuraDepartment of Pediatrics, Hematology and Oncology, Medical University of Gdańsk, Gdańsk, Poland.
Monika RadwańskaDepartment of Pediatric Oncology and Hematology, College of Medical Sciences, University of Rzeszów, Rzeszów, Poland.
Iwona RurańskaDepartment of Pediatrics, Hematology and Oncology, Medical University of Silesia, Katowice, Poland.
Tomasz SzczepańskiDepartment of Pediatrics, Hematology and Oncology, Medical University of Silesia, Katowice, Poland.
Dariusz WasińskiDepartment of Pediatric Hematology and Oncology, Collegium Medicum, University of Zielona Góra, Zielona Góra, Poland.
Irena Woźnica-KarczmarzClinic of Pediatric Hematology and Oncology, Medical University of Lublin, Lublin, Poland.
Karolina ZielezińskaDepartment of Pediatrics, Hemato-Oncology and Gastroenterology, Pomeranian Medical University, Szczecin, Poland.
Aleksandra KrólakDepartment of Pediatrics, Hemato-Oncology and Gastroenterology, Pomeranian Medical University, Szczecin, Poland.
Tomasz OciepaDepartment of Pediatrics, Hemato-Oncology and Gastroenterology, Pomeranian Medical University, Szczecin, Poland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: In 2020, the new nationwide protocol of prophylaxis in Polish plasma-derived FVIII (pdFVIII) previously treated patients (PTPs) with severe hemophilia A (sHA) was introduced, resulting in the necessity of switching from pdFVIII to recombinant FVIII (octocog-alpha; rFVIII). The study aimed to: (1) assess the safety of switching from pdFVIII to rFVIII, (2) assess the safety and efficacy of pharmacokinetically based (PK-based) personalized prophylaxis in severe hemophilia A. Patients and methods: 151 children and adolescents receiving prophylaxis with a standard dose (40 U/kg 3 x weekly) of pdFVIII were included in this study. Annualized bleeding rate (ABR) and annualized joint bleeding rate (AJBR) were analyzed for all patients before enrollment. Using myPKFiT application, pharmacokinetic (PK) analysis followed by the selection of the optimal model of prophylaxis was performed in all patients. Two possible models of prophylaxis (standard-dose rFVIII versus PK-based rFVIII) were discussed, with parents leaving the choice to their decision. Parents reported all episodes of bleeds. Screening for inhibitor was performed every 3 months. ABR and AJBR were prospectively analyzed again after a minimum follow-up time of 26 weeks. Results: 141/151 (93.4%) patients completed the study. 34 patients decided to continue standard prophylaxis with rFVIII (Group I), whereas 107 were switched to PK-based prophylaxis (Group II). The risk of inhibitor development could be assessed in 137/151 (90.7%) patients. Only 2/137 (1.47%) patients (both on PK-based prophylaxis) developed low-titer inhibitor with its spontaneous elimination. The retrospective analysis of bleeds during the last 12 months of standard pdFVIII prophylaxis revealed that patients who decided to continue standard prophylaxis had historically lower ABR and AJBR than those who started PK-based personalized prophylaxis. After a minimum of 26 weeks, ABR and AJBR improved significantly in both groups. There was no significant difference in ABR and AJBR between Group I and Group II during the follow-up period. However, the rate of reduction of ABR and AJBR was higher in patients on PK-based personalized prophylaxis. Conclusion: (1) Switching from pdFVIII to rFVIII (octocog-alpha) in PTPs with sHA is safe, (2) PK-based personalized prophylaxis may decrease ABR and AJBR in children and adolescents with sHA.

Indexed as

annualized bleeding rateannualized joint bleeding ratebleeding prophylaxischildrenhemophilia Aplasma-derived FVIIIrecombinant FVIII

Identifiers

PMID36911027
PMCPMC9996297

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