Tratamiento de la hemoglobinuria paroxística nocturna con eculizumab: experiencia en España

Rubio, Montserrat López; Morado, Marta; Gaya, Ana; Rosa, D Alonso; Ojeda, Emilio; Muñoz, Juan Antonio; Mendiguren, Begoña Pérez de; Monteagudo, María Dolores; Durán, J. M.; Fisac, Rosa; Moreno, Damián; Villegas, Ana

doi:10.1016/j.medcli.2010.09.035

Cited by 10 publications

(3 citation statements)

References 27 publications

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“…Fifteen real-life NRSI including individuals with PNH (n = 4189) and aHUS (n = 1090) were assessed mathematically into pairwise level. As observed in Figure 4, a protective effect from eculizumab was evident in hemolysis in PNH [28][29][30][31][32][33][34][35][36][37][38][39][40][41][42][43][44], TMA in aHUS , and AKI in aHUS As shown in Figure 5, eculizumab had a positive effect, respectively, in clinical trials and reallife NRSI, on thrombotic events in PNH (0.07/0.03 to 0.19, 0.24/0.17 to 0.33) and chronic kidney disease (CKD) occurrence/progression in PNH (0.31/0.10 to 0.97, 0.66/0.44 to 0.98). No (or non-important) heterogeneity and almost no (or weak) funnel plot asymmetry affected these calculations.…”

Section: Drug Intervention † C5 Inhibitorsmentioning

confidence: 62%

“…Fifteen real-life NRSI including individuals with PNH ( n = 4189) and aHUS ( n = 1090) were assessed mathematically into pairwise level. As observed in Figure 4 , a protective effect from eculizumab was evident in hemolysis in PNH [ 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 ], TMA in aHUS [ 53 , 54 , 55 , 56 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 , 65 , 66 , 67 , 68 , 69 , 70 , 71 , 72 , 73 , 74 , 75 , 76 ], and AKI in aHUS [ 53 , 54 , 55 , 56 , 57 , 58 , …”

Section: Resultsmentioning

confidence: 95%

“…After excluding clearly irrelevant studies (i.e., pre-clinical studies and clinical trials with no evaluation of the eligible outcomes, other observational studies that did not meet the conditions to be considered as real-life NRSI, such as case series or case reports), 28 pharmaceutical industry-sponsored clinical trials corresponding to phases 1 to 3 evaluation of various complement inhibitors, and 15 real-life NRSI reflecting uses of these medicines in real-world settings were found to be eligible: these studies assess outcomes in PNH (No. of clinical trials/real-life NRSI: 7/7), aHUS (7/8), rgMG (3/0), aAMR (6/0), and DGF (5/0), and included a total population of 7484 participants [ 17 , 18 , 19 , 20 , 21 , 22 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 55 , 56 , 57 , 58 , 59 ,…”

Section: Resultsmentioning

confidence: 99%

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The Inhibition of Complement System in Formal and Emerging Indications: Results from Parallel One-Stage Pairwise and Network Meta-Analyses of Clinical Trials and Real-Life Data Studies

et al. 2020

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This manuscript presents quantitative findings on the actual effectiveness of terminal complement component 5 (C5) inhibitors and complement component 1 (C1) esterase inhibitors through their formal and common “off-label” (compassionate) indications. The results emanated from pairwise and network meta-analyses to present evidence until September 2019. Clinical trials (CT) and real-life non-randomized studies of the effects of interventions (NRSI) are consistent on the benefits of C5 inhibitors and of the absence of effects of C1 esterase inhibitors (n = 7484): Mathematically, eculizumab (surface under the cumulative ranking area (SUCRA) >0.6) and ravulizumab (SUCRA ≥ 0.7) were similar in terms of their protective effect on hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), thrombotic microangiopathy (TMA) in atypical hemolytic uremic syndrome (aHUS), and acute kidney injury (AKI) in aHUS, in comparison to pre-/off-treatment state and/or placebo (SUCRA < 0.01), and eculizumab was efficacious on thrombotic events in PNH (odds ratio (OR)/95% confidence interval (95% CI) in CT and real-life NRSI, 0.07/0.03 to 0.19, 0.24/0.17 to 0.33) and chronic kidney disease (CKD) occurrence/progression in PNH (0.31/0.10 to 0.97, 0.66/0.44 to 0.98). In addition, meta-analysis on clinical trials shows that eculizumab mitigates a refractory generalized myasthenia gravis (rgMG) crisis (0.29/0.13 to 0.61) and prevents new acute antibody-mediated rejection (AMR) episodes in kidney transplant recipients (0.25/0.13 to 0.49). The update of findings from this meta-analysis will be useful to promote a better use of complement inhibitors, and to achieve personalization of treatments with this class of drugs.

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Section: Drug Intervention † C5 Inhibitorsmentioning

confidence: 62%

“…Fifteen real-life NRSI including individuals with PNH ( n = 4189) and aHUS ( n = 1090) were assessed mathematically into pairwise level. As observed in Figure 4 , a protective effect from eculizumab was evident in hemolysis in PNH [ 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 ], TMA in aHUS [ 53 , 54 , 55 , 56 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 , 65 , 66 , 67 , 68 , 69 , 70 , 71 , 72 , 73 , 74 , 75 , 76 ], and AKI in aHUS [ 53 , 54 , 55 , 56 , 57 , 58 , …”

Section: Resultsmentioning

confidence: 95%