Evidence map›Paper›PMID 33512400›Full record

ReviewBlood2021

How I treat paroxysmal nocturnal hemoglobinuria.

Robert A Brodsky

Abstract readCase ReportsReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Blood, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 92 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
92citing 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.

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

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

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32 more citing papers are in PubMed but not listed here.

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

1 author.

Robert A BrodskyDivision of Hematology, Johns Hopkins Medicine, Baltimore, MD.

Funding

Complementopathies: Genotype and PhenotypeR01HL133113 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI BRODSKY, ROBERT A · 2016 to 2020
$2.0M
NHLBI NIH HHS R01 HL133113
6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure, a thrombophilia, or any combination of the above. Terminal complement inhibition is highly effective for treating intravascular hemolysis from PNH and virtually eliminates the risk of thrombosis, but is not effective for treating bone marrow failure. Here, I present a variety of clinical vignettes that highlight the clinical heterogeneity of PNH and the attributes and limitations of the 2 US Food and Drug Administration-approved C5 inhibitors (eculizumab and ravulizumab) to treat PNH. I review the concept of pharmacokinetic and pharmacodynamic breakthrough hemolysis and briefly discuss new complement inhibitors upstream of C5 that are in clinical development. Last, I discuss the rare indications for bone marrow transplantation in patients with PNH.

Indexed as

AdultAntibodies, Monoclonal, HumanizedComplement Inactivating AgentsDisease ManagementDrug DevelopmentFemaleHemoglobinuria, ParoxysmalHemolysisHumansMaleMiddle AgedThrombosisAntibodies, Monoclonal, HumanizedComplement Inactivating Agentseculizumabravulizumab

Identifiers

PMID33512400
PMCPMC7955407

What Socratic holds

Textmetadata
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.