Eculizumab in low-middle income countries: how much does a life cost?

Nga, Hong Si; Palma, Lílian Monteiro Pereira; Neto, Miguel Ernandes; Andrade, Luís Gustavo Modelli de

doi:10.1007/s40620-022-01282-4

Cited by 5 publications

(6 citation statements)

References 7 publications

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“…More treated patients had history of TEs/MAVEs and HDA, indicating a more severe clinical presentation. While treatment recommendations support the use of eculizumab for patients with hemolytic PNH, eculizumab may not be readily accessible, such as in low‐middle income countries 1,20,21 …”

Section: Discussionmentioning

confidence: 99%

“…eculizumab for patients with hemolytic PNH, eculizumab may not be readily accessible, such as in low-middle income countries. 1,20,21 Previous studies showed survival rates of 95-100% at 5 years with eculizumab. 11,16,22 However, these previous studies had limitations with respect to the sample size, contemporaneous data for comparator populations, and duration of time on eculizumab.…”

Section: Discussionmentioning

confidence: 99%

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Long‐term effectiveness of eculizumab: Data from the International PNH Registry

Terriou,

Lee,

Forsyth

et al. 2023

European J of Haematology

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ObjectivesData from the International PNH Registry (NCT01374360) were used to estimate the overall survival and first occurrence of thromboembolic events/major adverse vascular events (TEs/MAVEs) for eculizumab‐treated patients with paroxysmal nocturnal hemoglobinuria (PNH) compared with a contemporaneous untreated cohort.MethodsPatients enrolled in the Registry from March 16, 2007, to February 14, 2022, were included. Treated patients received eculizumab for >35 days; untreated patients did not receive eculizumab at any time. Univariable and multivariable analyses were performed using a Cox proportional hazards regression model comparing eculizumab treatment periods to untreated periods and were adjusted for baseline covariates (e.g., high disease activity [HDA], transfusion dependency, and eculizumab treatment status).ResultsThe analysis included 4118 patients. The univariable hazard ratio (HR) (95% CI) for mortality in eculizumab‐treated time versus untreated time was 0.51 (0.41–0.64; p < 0.0001). Significant baseline covariates included age, sex, history of bone marrow failure, ≥4 erythrocyte transfusions within 12 months before baseline, and an estimated glomerular filtration rate ≤ 60 mL/min/1.73 m2 (all p < 0.0001). In the adjusted analysis, patients with baseline HDA had the greatest reduction in mortality risk (HR [95% CI], 0.51 [0.36–0.72]). Treated patients had approximately 60% reduction in TE/MAVE risk during treated versus untreated time (HR [95% CI]: TE: 0.40 [0.26–0.62], MAVE: 0.37 [0.26–0.54]; p < 0.0001).ConclusionUsing data from the largest Registry of patients with PNH, with ≥14 years of overall follow‐up, we demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE/MAVE risk.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Long‐term effectiveness of eculizumab: Data from the International PNH Registry

Terriou,

Lee,

Forsyth

et al. 2023

European J of Haematology

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“…To prevent chronic organ damage, it is of utmost importance to address the underlying etiology quickly. There is an urgent need to engage public and private health systems 22 in the diagnosis and protocols for early treatment according to the underlying cause.…”

Section: Why Is It So Important To Talk About Hemolytic Uremic Syndro...mentioning

confidence: 99%

We still need to talk about Hemolytic Uremic Syndrome: early recognition is key!

Palma

2023

Braz. J. Nephrol.

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“…In conclusion, despite revolutionizing the treatment of PNH with well-established efficacy and safety, eculizumab has not been established as the mainstay of treatment in Nepal's PNH patients. Moving forward, the acquisition of the drug by the public system could be the best way to combat the overpricing of the drug [25]. This could still turn out to be quite expensive per patient for low-and middle-income countries like Nepal, and in this regard, manufacturers must resort to price reduction strategies for such countries.…”

Section: Letter To the Editormentioning

confidence: 99%

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

Adhikari¹,

Sapkota

Shrestha

et al. 2023

Orphanet J Rare Dis

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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.

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Eculizumab in low-middle income countries: how much does a life cost?

Cited by 5 publications

References 7 publications

Long‐term effectiveness of eculizumab: Data from the International PNH Registry

Long‐term effectiveness of eculizumab: Data from the International PNH Registry

We still need to talk about Hemolytic Uremic Syndrome: early recognition is key!

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

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