Evidence map›Paper›PMID 33512711›Full record

ArticleBritish journal of clinical pharmacology2021

The potential of individualized dosing of ravulizumab to improve patient-friendliness of paroxysmal nocturnal haemoglobinuria treatment at reduced costs.

Mendy Ter Avest, Saskia M C Langemeijer, Saskia E M Schols, David M Burger, Nicole C A J van de Kar, Nicole M A Blijlevens, Wietske Kievit, Rob Ter Heine

Open access · hybridAbstract read
In one paragraph

Article in British journal of clinical pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
0.4field-weighted citation impact, top 44% of its field
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

6 citing papers in PubMed, 1 synthesis or guideline pooled it, 8 citations in OpenAlex.

  1. Guideline
  2. Article
  3. Observational
  4. Article
  5. Article
  6. 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

8 authors at 1 institution in 1 country.

Mendy Ter AvestDepartment of Pharmacy, Radboud Institute for Health Sciences, Radboud University Medical Centre, Nijmegen, The Netherlands.ORCID 0000-0001-5757-4919
Saskia M C LangemeijerDepartment of Hematology, Radboud University Medical Centre, Nijmegen, The Netherlands.
Saskia E M ScholsDepartment of Hematology, Radboud University Medical Centre, Nijmegen, The Netherlands.
David M BurgerDepartment of Pharmacy, Radboud Institute for Health Sciences, Radboud University Medical Centre, Nijmegen, The Netherlands.ORCID 0000-0001-7279-2818
Nicole C A J van de KarDeparment of Pediatric Nephrology, Radboud University Medical Centre, Nijmegen, The Netherlands.
Nicole M A BlijlevensDepartment of Hematology, Radboud University Medical Centre, Nijmegen, The Netherlands.
Wietske KievitDepartment for Health Evidence, Radboud University Medical Centre, Nijmegen, the Netherlands.
Rob Ter HeineDepartment of Pharmacy, Radboud Institute for Health Sciences, Radboud University Medical Centre, Nijmegen, The Netherlands.ORCID 0000-0003-2185-8201
Radboud University Nijmegen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Ravulizumab is a very expensive complement C5-inhibitor for the treatment of paroxysmal nocturnal haemoglobinuria, with a fixed-dosing interval of 8 weeks. For lifelong treatment, a cost-effective and patient-friendly dosing strategy is preferred. We therefore explored alternative ravulizumab dosing regimens in silico based on the thorough dose-finding studies of the manufacturer. Extending the interval to 10 weeks or individually extending the interval to a mean of 12.8 weeks based on pharmacokinetic monitoring resulted in noninferior efficacy in terms of lactate dehydrogenase normalization, with drug cost savings up to 37%. We here show the potential of individualized ravulizumab dosing to improve patient-friendliness at reduced costs.

Indexed as

Hemoglobinuria, ParoxysmalAntibodies, Monoclonal, HumanizedComplement Inactivating AgentsCost-Benefit AnalysisHumansAntibodies, Monoclonal, HumanizedComplement Inactivating Agentsravulizumabparoxysmal nocturnal haemoglobinuriapharmacodynamicspharmacokineticsravulizumab

Identifiers

PMID33512711
PMCPMC8359320
OpenAlexW3124759748

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.