Evaluación del tratamiento sustitutivo del enfisema por déficit de alfa-1-antitripsina

Miravitlles, Marc; Vidal, Rafael; Torrella, M.; Bofill, J M; Gracia, Javier de; Cotrina, Montserrat

doi:10.1016/s0300-2896(15)30997-2

Cited by 26 publications

(10 citation statements)

References 20 publications

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“…Biochemical efficacy at the higher dose given monthly did not demonstrate a protective AAT level in 3 of 16 patients 50. A second study investigating the pharmacokinetics of an alternative dose (Prolastin, 120 mg/kg every 2 weeks) was completed in 23 participants;46 none of the patients maintained target serum AAT levels (>80 mg/dL) and only two maintained serum AAT levels >70 mg/dL from days 7 to 14 suggesting insufficient dosing.…”

Section: Resultsmentioning

confidence: 97%

“…Pulmonary function was reported in five studies with no significant changes in rates of decline or spirometry being observed 46,47,50–52. An observational registry study where longitudinal FEV 1 follow-up was available in 287 patients over 37.8±18.9 months showed significant differences in decline in FEV 1 <30% predicted and FEV 1 30%–65% groups (−35.6±21.3 vs −64.0±26.4 mL; P =0.0008) 52…”

Section: Resultsmentioning

confidence: 99%

“…Twelve uncontrolled observational studies using intravenous AAT augmentation in 2,526 patients were included, which assessed safety and tolerability,43–45 pharmacokinetics,46–48 pulmonary neutrophilic inflammation,49 longitudinal change in lung function,50–52 and clinical characteristics or recipients 53,54. AAT augmentation was reported as safe and well tolerated (one study drug-related AE out of 555 doses)45 with 26 AEs probably/possibly related to the study drug,43,45,47 largely comprising symptoms commonly observed in infusions of other protein-based products 52.…”

Section: Resultsmentioning

confidence: 99%

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Treatment of lung disease in alpha-1 antitrypsin deficiency: a systematic review

Edgar¹,

Patel²,

Bayliss³

et al. 2017

COPD

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BackgroundAlpha-1 antitrypsin deficiency (AATD) is a rare genetic condition predisposing individuals to chronic obstructive pulmonary disease (COPD). The treatment is generally extrapolated from COPD unrelated to AATD; however, most COPD trials exclude AATD patients; thus, this study sought to systematically review AATD-specific literature to assist evidence-based patient management.MethodsStandard review methodology was used with meta-analysis and narrative synthesis (PROSPERO-CRD42015019354). Eligible studies were those of any treatment used in severe AATD. Randomized controlled trials (RCTs) were the primary focus; however, case series and uncontrolled studies were eligible. All studies had ≥10 participants receiving treatment or usual care, with baseline and follow-up data (>3 months). Risk of bias was assessed appropriately according to study methodology.ResultsIn all, 7,296 studies were retrieved from searches; 52 trials with 5,632 participants met the inclusion criteria, of which 26 studies involved alpha-1 antitrypsin augmentation and 17 concerned surgical treatments (largely transplantation). Studies were grouped into four management themes: COPD medical, COPD surgical, AATD specific, and other treatments. Computed tomography (CT) density, forced expiratory volume in 1 s, diffusing capacity of the lungs for carbon monoxide, health status, and exacerbation rates were frequently used as outcomes. Meta-analyses were only possible for RCTs of intravenous augmentation, which slowed progression of emphysema measured by CT density change, 0.79 g/L/year versus placebo (P=0.002), and associated with a small increase in exacerbations 0.29/year (P=0.02). Mortality following lung transplant was comparable between AATD- and non-AATD-related COPD. Surgical reduction of lung volume demonstrated inferior outcomes compared with non-AATD-related emphysema.ConclusionIntravenous augmentation remains the only disease-specific therapy in AATD and there is evidence that this slows decline in emphysema determined by CT density. There is paucity of data around other treatments in AATD. Treatments for usual COPD may not be as efficacious in AATD, and further studies may be required for this disease group.

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Section: Resultsmentioning

confidence: 97%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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Treatment of lung disease in alpha-1 antitrypsin deficiency: a systematic review

Edgar¹,

Patel²,

Bayliss³

et al. 2017

COPD

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“…Hoy día todavía se considera en algunas normativas que sólo se debe sospechar el déficit en pacientes con enfisema grave, jóvenes y/o no fumadores 75 ; sin embargo, estos individuos son sólo una pequeña parte de todos los casos y muchos pacientes no encajan en estos parámetros de gravedad 45 . En un estudio realizado en España, el retraso en el diagnóstico, medido como el tiempo entre el diagnóstico de la EPOC y el del déficit de AAT, fue de un promedio de 10 años 76 .…”

Section: Detección De Casos De Déficit De Alfa-1-antitripsina ¿Por Qunclassified

Déficit de alfa-1-antitripsina. Situación en España y desarrollo de un programa de detección de casos

Roza

Lara

Vilà

et al. 2006

Archivos de Bronconeumología

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“…The characteristics of these patients have been described elsewhere. 16 This treatment was discontinued 1 month before the study as well as during the study period. Patients with bronchiectasis showed more preserved pulmonary function, represented by higher FVC, FEV 1 , FEV 1 /FVC, and diffusion of carbon monoxide corrected for alveolar volume, and lower values of total lung capacity and residual volume than patients without bronchiectasis, with the majority of them having pulmonary emphysema with greater airflow obstruction, air trapping, and decreased diffusing capacity (Table 2).…”

Section: Patient Characteristicsmentioning

confidence: 99%

Specific Antibody Response Against the 23-Valent Pneumococcal Vaccine in Patients With α1-Antitrypsin Deficiency With and Without Bronchiectasis

Miravitlles

Gracia

Rodrigo³

et al. 1999

Chest

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Evaluación del tratamiento sustitutivo del enfisema por déficit de alfa-1-antitripsina

Cited by 26 publications

References 20 publications

Treatment of lung disease in alpha-1 antitrypsin deficiency: a systematic review

Treatment of lung disease in alpha-1 antitrypsin deficiency: a systematic review

Déficit de alfa-1-antitripsina. Situación en España y desarrollo de un programa de detección de casos

Specific Antibody Response Against the 23-Valent Pneumococcal Vaccine in Patients With α1-Antitrypsin Deficiency With and Without Bronchiectasis

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