Evidence map›Paper›PMID 39235489›Full record

ReviewAnnals of hematology2025

Clinical characteristics and management of paroxysmal nocturnal haemoglobinuria in Latin America: a narrative review.

Valentina Goldschmidt, Elia Ixel Apodaca, Kenny Mauricio Gálvez, Bruno Wannesson, Phillip Scheinberg

Abstract readReview
In one paragraph

Review in Annals of hematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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.

Valentina GoldschmidtHematology Department, Hospital Padre Hurtado, Esperanza 2150, San Ramon, 8880465, Santiago, Chile. hematologia.vg@gmail.com.
Elia Ixel ApodacaHematology and Oncology Department, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
Kenny Mauricio GálvezCancer Unit, Hospital Pablo Tobón Uribe, Medellín, Colombia.
Bruno WannessonHematology Department, Fundaleu, Buenos Aires, Argentina.
Phillip ScheinbergDivision of Hematology, Hospital A Beneficiência Portuguesa, São Paulo, Brazil.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, complement-associated, haematological disorder. The level of knowledge about the disease and its management varies around the world. This narrative review provides an overview of available clinical data on PNH in Latin America (LATAM). A search of the PubMed, EMBASE and LILACS/IBECS databases to February 2023, and addition of author-known articles, yielded 24 relevant published articles, the majority (n = 15) from Brazil. Fourteen articles were full papers; 10 were conference abstracts. The prevalence of PNH in Brazil is estimated at 1:237,000 inhabitants. Among blood samples sent for flow cytometry screening for suspected PNH in Brazil and Colombia, 14 - 30% were positive. There is suggestion that disease subtypes may differ among LATAM populations, with classical PNH more common in Brazilian patients and PNH with aplastic anaemia more common in Mexican patients. Median age at diagnosis of PNH ranged from 24 to 41 years. Common symptoms included fatigue, haemoglobinuria, and abdominal pain, although the symptom profile varied by subtype. Three available studies indicated that eculizumab was effective at reducing haemolysis, improving anaemia, and reducing the risk of thrombosis in patients with PNH with intravascular haemolysis. A consensus document from the Brazilian Association of Hematology, Hemotherapy and Cell Therapy RBC and Iron Committee provides guidance on identifying and managing PNH patients, including appropriate selection of patients for eculizumab. Additional data on the epidemiology, natural history and outcomes of patients with PNH in LATAM countries are needed to better understand the disease and its management throughout the region.

Indexed as

Hemoglobinuria, ParoxysmalAdultAntibodies, Monoclonal, HumanizedDisease ManagementHemolysisHumansLatin AmericaPrevalenceAntibodies, Monoclonal, HumanizedeculizumabComplement inhibitionEculizumabLatin AmericaNarrative reviewParoxysmal nocturnal haemoglobinuria

Identifiers

PMID39235489
PMCPMC11971169

What Socratic holds

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LicenceCC BY-NC-ND
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Registered trials

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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.