PMID- 25938295
OWN - NLM
STAT- MEDLINE
DCOM- 20160125
LR  - 20161125
IS  - 1541-2563 (Electronic)
IS  - 1541-2563 (Linking)
VI  - 12 Suppl 1
DP  - 2015 May
TI  - Antitrypsin Deficiency Assessment and Programme for Treatment (ADAPT): The United 
      Kingdom Registry.
PG  - 63-8
LID - 10.3109/15412555.2015.1021911 [doi]
AB  - The study of rare diseases is compromised by its rarity. The establishment of 
      national and international registries can overcome many of the problems and be 
      used for many monogenetic conditions with relatively consistent outcomes and thus 
      lead to a consistency of clinical management by centres of excellence. However, 
      in Alpha-1 antitrypsin deficiency (AATD), the outcome is highly variable in terms 
      of the organ(s) most affected and the diversity of disease penetration and 
      progression. This creates the added difficulty of understanding the disease 
      sufficiently to monitor and advise the patients to the standard required and 
      importantly design and deliver clinical trials that address the many facets of 
      the disease. The development of research registries and centres of excellence 
      provides the necessary expertise and data to further the understanding and 
      management of diseases like AATD though with significant cost implications. The 
      ADAPT programme was established in 1996 with extensive core funding to enable 
      patients to be seen from all regions of the United Kingdom as an addition to the 
      National Health Service without appointment time constraints and with the purpose 
      of collecting extensive state of the art demographics. The model has proven to be 
      highly productive providing new insights especially into the lung disease, 
      generating and delivering on clinical trials and importantly establishing active 
      patient groups and participation.
FAU - Stockley, Robert A
AU  - Stockley RA
AD  - ADAPT Project, Queen Elizabeth Hospital Birmingham , Birmingham , United Kingdom.
LA  - eng
PT  - Journal Article
PL  - England
TA  - COPD
JT  - COPD
JID - 101211769
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Disease Progression
MH  - Female
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Phenotype
MH  - *Registries
MH  - United Kingdom
MH  - Young Adult
MH  - alpha 1-Antitrypsin Deficiency/diagnosis/genetics/*therapy
OTO - NOTNLM
OT  - alpha-1 antitrypsin
OT  - deficiency
OT  - demographics
OT  - emphysema
OT  - liver disease
OT  - registry
EDAT- 2015/05/06 06:00
MHDA- 2016/01/26 06:00
CRDT- 2015/05/05 06:00
PHST- 2015/05/05 06:00 [entrez]
PHST- 2015/05/06 06:00 [pubmed]
PHST- 2016/01/26 06:00 [medline]
AID - 10.3109/15412555.2015.1021911 [doi]
PST - ppublish
SO  - COPD. 2015 May;12 Suppl 1:63-8. doi: 10.3109/15412555.2015.1021911.