Analysis of baseline characteristics, disease burden and long-term follow-up of 167 patients with Paroxysmal Nocturnal Hemoglobinuria at a single center in Brazil

Azambuja, Ana Paula de; Oliveira, Marcela M.; Bitencourt, Marco A.; Bonfim, Carmem; Malvezzi, Mariester; Pasqüini, Ricardo

doi:10.1016/j.bcmd.2021.102605

Cited by 4 publications

(2 citation statements)

References 24 publications

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“…At the 2019 American Society of Hematology annual meeting, Yamakawa et al presented data from 109 patients with a PNH clone treated at two centers in the city of São Paulo, SP, of which 56 had hemolytic disease [ 22 ]. In 2021, de Azambuja et al reported a retrospective cohort of 167 PNH patients and the impact of disease burden on long-term follow-up [ 23 ]. Pires da Silva et al published a retrospective series of 87 patients with PNH detected by flow cytometry between 2006 and 2019 at a single Brazilian referral center, highlighting the highly variable clinical presentation of the disease [ 24 ].…”

Section: Discussionmentioning

confidence: 99%

Characteristics of paroxysmal nocturnal hemoglobinuria patients in Brazil: A retrospective administrative claims database analysis of PNH patients in Brazilian public healthcare system

Menosi Gualandro,

Salvino,

Bassolli de Oliveira Alves

et al. 2023

PLoS ONE

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Introduction Few studies have reported the profile of patients with paroxysmal nocturnal hemoglobinuria (PNH) and their care in the Brazilian health system. Objective To describe clinical and epidemiological characteristics of patients with PNH in the Brazilian public health system including procedures performed, associated comorbidities and visits to health care professionals. Methods In a real-world observational, retrospective, population-based cohort study, anonymized secondary data provided by the Department of Informatics of the Brazilian Unified Health System (DATASUS) were analyzed. Patients were considered eligible if they had at least one procedure coded with the ICD-10 code D59.5 from January 1, 2008 to December 30, 2018. Results In total, 675 individual PNH patients were identified (52.4% female; prevalence of 1:237,000 people). Around 15.8% of the patients included had myelodysplastic syndrome and about half of the sample had other aplastic anemias and/or other bone marrow failure syndromes. Portal vein thrombosis (I82 ICD code) was reported in 4.3% of patients. Regarding hospitalizations, 263 individual PNH patients had 416 inpatient admissions with the ICD code for PNH (D59.5) on admission. Twelve deaths occurred during the study period, of which two had the PNH ICD code related with the cause of death, while another three deaths were associated with acquired hemolytic anemia (D59.9), unspecified aplastic anemia (D61.9) and acute respiratory failure (J96.0), respectively. Conclusion Despite its limitations, this statistical analysis of data extracted from DATASUS reasonably describes PNH patients in Brazil and its variations across different regions of the country. Comorbidities frequently associated with PNH such as portal vein thrombosis were not as common in our study, but it is assumed that several thrombotic events at specific sites were coded under the broader I82 ICD code. The frequency of visits to different health professionals, including hematologists, increased after the diagnosis of PNH. Among hospitalized PNH patients, the mortality rate was 4.5%.

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Section: Discussionmentioning

confidence: 99%

Characteristics of paroxysmal nocturnal hemoglobinuria patients in Brazil: A retrospective administrative claims database analysis of PNH patients in Brazilian public healthcare system

Menosi Gualandro,

Salvino,

Bassolli de Oliveira Alves

et al. 2023

PLoS ONE

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“…31,32 The GPI deficiency on all or most mature peripheral blood cells allows rapid diagnosis of PNH via flow cytometry 33 even in resource-poor settings, if clinical suspicion settings exists. 34,35 However, diagnosis of PNH is unfortunately still delayed with time from occurrence of first disease-specific symptoms until final diagnosis too often ranging from months to even years, [36][37][38] even in patients with preceding AA or low-risk MDS, who need to be monitored enduringly for the occurrence and development of PNH after successful treatment of their primary BMF manifestation. 39,40…”

Section: Pnh-we Know the Cause While We Do Not Finally Know What Is C...mentioning

confidence: 99%

Paroxysmal nocturnal hemoglobinuria: Where we stand

Panse

2023

American J Hematol

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For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied-up until recently-on antibody based terminal complement inhibitionon. PNH pathophysiology-a mutational defect leading to partial or complete absence of complement-regulatory proteins on blood cells-leads to intravascular hemolysis and consequences such as thrombosis and other sequelae. A plethora of new drugs interfering with the proximal and terminal complement cascade are under recent development and the first "proof-of-pinciple" proximal complement inhibitor targeting C3 has been approved in 2021. "PNH: where we stand" will try to give a brief account on where we came from and where we stand focusing on approved therapeutic options. The associated improvements as well as potential consequences of actual and future treatments as well as their impact on the disease will continue to necessitate academic and scientific focus on improving treatment options as well as on side effects and outcomes relevant to individual patient lives and circumstances in order to develop effective, safe, and available treatment for all hemolytic PNH patients globally.Note: Due to space limitation the author mostly cites reviews and overviews, giving readers the chance to easily find continuative summaries of potentially interesting topics. He therefore does apologize

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Efficacy and safety of current treatments for paroxysmal nocturnal hemoglobinuria: A systematic review

Pires

Bonetti

Ciecilinsky

et al. 2023

Clinical Immunology Communications

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Analysis of baseline characteristics, disease burden and long-term follow-up of 167 patients with Paroxysmal Nocturnal Hemoglobinuria at a single center in Brazil

Cited by 4 publications

References 24 publications

Characteristics of paroxysmal nocturnal hemoglobinuria patients in Brazil: A retrospective administrative claims database analysis of PNH patients in Brazilian public healthcare system

Characteristics of paroxysmal nocturnal hemoglobinuria patients in Brazil: A retrospective administrative claims database analysis of PNH patients in Brazilian public healthcare system

Paroxysmal nocturnal hemoglobinuria: Where we stand

Efficacy and safety of current treatments for paroxysmal nocturnal hemoglobinuria: A systematic review

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