Evidence map›Paper›PMID 34275086›Full record

ArticleAdvances in therapy2021

Real-World Healthcare Resource Utilization (HRU) and Costs of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Receiving Eculizumab in a US Population.

Wendy Y Cheng, Sujata P Sarda, Nikita Mody-Patel, Sangeeta Krishnan, Mihran Yenikomshian, Malena Mahendran, Dominique Lejeune, Louise H Yu, Mei Sheng Duh

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed, 1 pooled it
2.0field-weighted citation impact, top 13% 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

22 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.

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  4. [How I diagnose and treat paroxysmal nocturnal hemoglobinuria].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2026
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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

9 authors at 3 institutions in 2 countries.

Wendy Y ChengAnalysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA, 02199-7668, USA. wendy.cheng@analysisgroup.com.ORCID http://orcid.org/0000-0002-8281-2496
Sujata P SardaApellis Pharmaceuticals, Inc., 100 5th Avenue, Waltham, MA, 02451, USA.
Nikita Mody-PatelApellis Pharmaceuticals, Inc., 100 5th Avenue, Waltham, MA, 02451, USA.
Sangeeta KrishnanApellis Pharmaceuticals, Inc., 100 5th Avenue, Waltham, MA, 02451, USA.
Mihran YenikomshianAnalysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA, 02199-7668, USA.
Malena MahendranGroupe d'analyse, Ltée, 1190 avenue des Canadiens-de-Montréal, Montréal, QC, H3B 0G7, Canada.
Dominique LejeuneGroupe d'analyse, Ltée, 1190 avenue des Canadiens-de-Montréal, Montréal, QC, H3B 0G7, Canada.
Louise H YuAnalysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA, 02199-7668, USA.
Mei Sheng DuhAnalysis Group, Inc., 111 Huntington Avenue, 14th Floor, Boston, MA, 02199-7668, USA.
Analysis Group (United States) · USApellis Pharmaceuticals (United States) · USGroup for Research in Decision Analysis · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionTo evaluate the economic burden and treatment patterns of patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with eculizumab, a C5 inhibitor, who were defined as blood transfusion-dependent (TD) versus blood transfusion-free (TF) in the US population.

methodsPatients aged at least 12 years with at least two claims for eculizumab infusion (first claim was the index date) were identified from the IBM

resultsOf 151 patients in the overall cohort (mean age 36.7 years; 55.6% female), 55 were TD (mean age 35.1 years; 67.3% female) and 96 were TF (mean age 37.6 years; 49.0% female). A total of 61% of patients (TD, 66%; TF, 58%) discontinued eculizumab, with TD patients having a shorter median time to discontinuation (TD, 0.5 years; TF, 0.9 years). TD patients had more all-cause hospitalizations than TF patients (p < 0.05). TD patients incurred higher all-cause direct medical costs (adjusted cost difference = $247,848) and medical-related absenteeism costs (adjusted cost difference = $4186) than TF patients (all p < 0.05), largely driven by hospitalizations. Similar trends were observed for PNH-related HRU and costs.

conclusionsThe economic burden of patients with PNH treated with eculizumab is greater among those dependent on blood transfusions.

Indexed as

Hemoglobinuria, ParoxysmalAdultAntibodies, Monoclonal, HumanizedDatabases, FactualFemaleHumansMalePatient Acceptance of Health CareAntibodies, Monoclonal, HumanizedeculizumabAbsenteeismBlood transfusionEconomic burdenEculizumabHealthcare resource utilizationMedical costsParoxysmal nocturnal hemoglobinuriaRetrospective studyTreatment patterns

Identifiers

PMID34275086
PMCPMC8342328
OpenAlexW3184889104

What Socratic holds

Textmetadata
LicenceCC BY-NC
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 Socratic graph.