Evidence map›Paper›PMID 37391775›Full record

ArticleOrphanet journal of rare diseases2023

Eculizumab for paroxysmal nocturnal haemoglobinuria: catastrophic health expenditure in Nepalese patients.

Sugat Adhikari, Surendra Sapkota, Suraj Shrestha, Kshitiz Karki, Anjan Shrestha

Abstract readLetter
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Trial
  2. Article
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

5 authors.

Sugat AdhikariShreegaun Primary Health Care Center, Dang, Nepal.
Surendra SapkotaDepartment of Internal Medicine, Ascension Saint Agnes Hospital, Baltimore, MD, USA.
Suraj ShresthaMaharajgunj Medical College, Institute of Medicine, Kathmandu, Nepal. multisurazz@gmail.com.ORCID 0000-0001-6888-260X
Kshitiz KarkiAnnapurna Neurological Institute and Allied Sciences, Kathmandu, Nepal.
Anjan ShresthaDepartment of Hematology, Tribhuvan University Teaching Hospital, Kathmandu, Nepal.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) results from a mutation in the phosphatidylinositol glycan class-A gene which causes uncontrolled complement activation with resultant intravascular hemolysis and its sequelae. Eculizumab is a terminal complement inhibitor that blocks this complement activation and has revolutionized the treatment of PNH but comes with an enormous price which can have catastrophic health expenditure in low-middle income countries (LMIC) like Nepal. Here, we discuss the potential way forwards in the treatment of PNH in Nepal and other LMICs.

Indexed as

Hemoglobinuria, ParoxysmalAntibodies, Monoclonal, HumanizedHealth ExpendituresHumansNepalAntibodies, Monoclonal, HumanizedeculizumabCatastrophic health expenditureEculizumabLow and middle-income countriesNepalOrphan drugParoxysmal nocturnal hemoglobinuria

Identifiers

PMID37391775
PMCPMC10311788

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.