Replacement therapy for alpha-1-protease inhibitor deficiency in PiZ subjects with chronic obstructive lung disease

Schmidt, E. W.; Rasche, B.; Ulmer, W. T.; Konietzko, N.; Becker, Michael; Lorenz, Joachim; Ferlinz, R

doi:10.1016/s0002-9343(88)80072-x

Cited by 28 publications

(15 citation statements)

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“…Taking into account the poor prognosis of patients with severe pulmonary emphysema due to α 1 -AT deficiency and the preliminary, yet convincing, data for α 1 -AT replacement therapy [7,[24][25][26][27], the study group felt that for ethical reasons it was not feasible to subject patients to a long-term i.v. placebo (e.g.…”

Section: Rationale and Efficacy Of α 1 -At Replacement Therapymentioning

confidence: 99%

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Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group

Wencker¹,

Banik²,

Buhl³

et al. 1998

Eur Respir J

118

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Alpha1-antitrypsin (alpha1-AT) deficiency is a genetic disorder characterized by low serum levels of alpha1-AT and a high risk of pulmonary emphysema at a young age. The resulting surplus of proteases, mainly of neutrophil elastase, can be balanced by i.v. augmentation with alpha1-AT. However, it is not clear if affected patients benefit from long-term augmentation therapy and no long-term safety data are available. We examined 443 patients with severe alpha1-AT deficiency and pulmonary emphysema receiving weekly i.v. infusions of 60 mg x kg body weight(-1) alpha1-AT in addition to their regular medication. The progression of the disease was assessed by repeated lung function measurements, particularly the decline in forced expiratory volume in one second (deltaFEV1). Four hundred and forty three patients with alpha1-AT deficiency tolerated augmentation therapy well with few adverse reactions. The deltaFEV1 in 287 patients with available follow-up data was 57.1+/-31.1 mL x yr(-1). Stratified for baseline FEV1, the decline was 35.6+/-21.3 mL in the 108 patients with an initial FEV1 <30% and 64.0+/-26.4 mL in the 164 with FEV1 30-65% of predicted normal (p=0.0008). The remaining 15 patients had an initial FEV1 >65% pred. Long-term treatment with i.v. alpha1-antitrypsin in patients with severe alpha1-antitrypsin deficiency is feasible and safe. The decline in forced expiratory volume in one second is related to the initial forced expiratory volume in one second as in alpha1-antitrypsin deficient patients not receiving augmentation therapy.

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Section: Rationale and Efficacy Of α 1 -At Replacement Therapymentioning

confidence: 99%

“…For short-term i.v. therapy with human plasma-derived α 1 -AT, safety and biochemical efficacy was demonstrated [7][8][9]. However, although several thousand patients receive augmentation therapy on an regular basis, data on safety and tolerability of long-term treatment with α 1 -AT is only anecdotal [10,11].…”

mentioning

confidence: 99%

Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group

Wencker¹,

Banik²,

Buhl³

et al. 1998

Eur Respir J

118

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“…Others have described adverse reactions, including hypoxaemia/cyanosis, hypotension, chest or back pain, haematoma-like spots, weight loss, transient leukocytosis, eosinophilia, fever, light-headedness, vertigo/dizziness, headaches and fatigue. The side-effects were self-limited, and none required hospitalization or alteration of the treatment [10][11][12][13][14]. In a series of animal studies using rabbits, monkeys, mice and rats, neither acute nor subacute toxicity studies presented any significant adverse effects [15].…”

Section: Discussionmentioning

confidence: 99%

Acute allergic reaction and demonstration of specific IgE antibodies against alpha-1-protease inhibitor

Meyer

Wencker²,

Teschler³

et al. 1998

Eur Respir J

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A 44 yr-old female with severe pulmonary emphysema and reduced alpha-1-protease inhibitor (alpha1-PI) serum levels developed an acute anaphylactic reaction following the third intravenous infusion of human alpha1-PI which was administered to prevent the progression of pulmonary emphysema. Specific immunoglobulin E-antibodies against human alpha1-PI could be demonstrated in the patient's serum using an enzyme allergosorbent test. Because of the risk of further severe anaphylactic reaction, the replacement therapy with alpha1-PI was discontinued. Physicians should be aware of this rare complication.

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“…[39][40][41][42][43] Infusions increase levels of AAT in broncho-alveolar lavage fluid, and the AAT recovered from the broncho-alveolar lavage is functionally active. Prolastin infusions appear to substantially correct the biochemical deficiency in Pi Z individuals.…”

Section: Augmentation Therapymentioning

confidence: 99%

Liver Disease in Alpha-1 Antitrypsin Deficiency

Miller¹

2015

JLRDT

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Replacement therapy for alpha-1-protease inhibitor deficiency in PiZ subjects with chronic obstructive lung disease

Cited by 28 publications

References 1 publication

Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group

Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group

Acute allergic reaction and demonstration of specific IgE antibodies against alpha-1-protease inhibitor

Liver Disease in Alpha-1 Antitrypsin Deficiency

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