Chest
Opinions/HypothesesAugmentation Therapy Reduces Frequency of Lung Infections in Antitrypsin Deficiency: A New Hypothesis With Supporting Data
Section snippets
Patients
Patients were recruited through announcements on theα1 Internet List. The appropriatequestionnaires were made available to all participants on the list andwere then returned by the responders by private e-mail to one personwho sorted the replies and forwarded them to Dr. Lieberman forevaluation.
Questionnaires
The questionnaires prepared for ZZ patients who wereparticipants on the α1 Internet List includedinformation on sex, age, and age when patients had received a diagnosisof AAT, as well as on smoking
Results
Questionnaires were filled out and submitted by 96 adult patientswith homozygous ZZ AAT deficiency (this includes one patient of SZphenotype) who were receiving α1-PI replacementtherapy (group I). This group included 50 men and 46 women. The agerange for men was 36 to 67 years (median age, 50 years), and the agerange for women was 33 to 72 years (median age, 53 years). The agerange at which a diagnosis of AAT deficiency was made for men was 28 to66 years (median, 40 years), and the age range
Discussion
The results of studies conducted by means of a questionnairesurvey directed to patients with AAT deficiency, who had ZZ phenotypeand were participating in the α1 Internet List,suggest that augmentation therapy with α1-PI isassociated with a marked reduction in the frequency of lung infectionsin the majority of patients. Most patients reported a frequency ofthree to five infections per year before startingα1-PI therapy, which dropped to zero to oneinfection per year while receiving α1-PI. Intwo
Questionnaire Used for Group I Patients Receivingα1-PI Augmentation Therapy
Questions for group II patients are the same as those for groupI, except for questions dealing with α1-PI. Thequestions that were eliminated were Nos. 4, 5, 6, 11a, 11b, 11c, 12a,12b, and 12c. Questions added were Nos. 11 and 12.
- 1.
Sex
- 2.
Age
- 3.
Age when diagnosed AAT deficient
- 4.
Date started on α1-PI
- 5.
How often do you receive α1-PIinfusion?
- 6.
How do you receive the infusion (hospital, doctor's office,home, other)?
- 7.
In addition to emphysema, do you have asthma?
- 8.
Do you have allergies?
- 9.
Do you have chronic bronchitis or
ACKNOWLEDGMENT
The author acknowledges the most valuableassistance of Mr. Carsten Larsen of Copenhagen, Denmark, and Mr. ClaudeBaril of Montreal, Canada in conducting the questionnaire survey. Thereport is dedicated to the memory of Mr. Claude Baril who was theoriginator of the α1 Internet List, which providesmuch-needed support to those born with AAT deficiency with theirpredisposition to lung and liver disease. His death on January 4, 1999,followed soon after a lung transplant operation.
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