abstract
144. Comorbidities in the Young and Aging
Populations with Haemophilia A
Ceri Hirst and James Black
Real World Data Science, F. Hoffmann La Roche,
Basel, Switzerland
Background: Haemophilia A (HA), a rare hereditary
condition, impacts ~1/5000 male births globally.
Treatment advances in recent decades have improved
life expectancy for affected individuals, now ap-
proaching normality. Thus, an aging population of
people with HA (PWHA) is emerging, but with little
published research on their comorbidity prole.
Objectives: We evaluated comorbidity rates from real
world data sources to show key comorbidities in
PWHA by age strata.
Methods: Analyses used 3 data sources, and their con-
cordance was evaluated. Clinical Practice Research
Database (CPRD) provides a comprehensive record
from a nationwide representative sample of UK gen-
eral practitioners. MarketScan provides insurance
claims data for ~46 million US patients. Patients from
CPRD and MarketScan were included if they had a
HA diagnosis on/before 1 Jan 2014 (index date), with
12 mo of medical history. An age- and gender-
matched sample of the general population was derived
in the CPRD. Diagnoses for 5 years preceding were
evaluated. A cross-sectional physician survey,
CHESS, collected data for EU patients with severe
HA.
Results: The population included MarketScan 1903,
CPRD 310, CHESS 996; mean age was 39 y (CPRD
and MarketScan) and 36 y (CHESS). In adults, hepati-
tis C, pain, arthropathy, chronic pulmonary disease
(including asthma), diabetes, cancer and anxiety/de-
pression appeared to be reported more often in PWHA
than the general population, though prevalence was in-
consistent across data sources. Prevalence of diabetes,
pain, depression/anxiety increased with age, while
HIV and hepatitis C prevalence was highest in 30-40
y group (likely due to childhood exposure to contami-
nated blood products). Arthropathy was reported in
MarketScan in PWHA aged 3-5 y (8%) and 6-11 y
(27%), but not CPRD, maybe due to lack of reporting
in primary care.
Conclusions: PWHA, across age groups, suffer con-
siderable comorbidities. Management of PWHA
Copyright 2016 John Wiley & Sons, Ltd.
89
should consider the likely higher prevalence of dis-
eases such as diabetes, mental health issues, pain, ar-
thropathy and asthma. Use of different types of data
source provides a comprehensive overview that may
enhance the recognition of comorbidities.
145. Epidemiologic and Clinical Characteristics of
Thalassemia (THAL) Intermedia (TI) in the US
Elliott Vichinsky1, Janet L. Kwiatkowski2, Patricia-
Jane Giardina3, Carole Paley4, Francis Vekeman5,
Wendy Y. Cheng6, Joseph Damron6, Nora
McCormick6, Medha Sasane4, Ying Qiu4, Mei Sheng
Duh6 and Alexis Thompson7
1
Childrens Hospital and Research Center Oakland,
Oakland; 2 The Childrens Hospital of Philadelphia,
Philadelphia; 3 Weill Cornell Medical College, New
York; 4 Novartis Pharmaceuticals Corporation, East
Hanover; 5 Groupe dAnalyse, Ltee, Montreal; 6 Analy-
sis Group, Inc., Boston; 7 Ann Robert H. Lurie Chil-
drens Hospital of Chicago, Chicago
Background: TI is increasingly prevalent in the US
due to changing immigration patterns. It is
underdiagnosed, leading to inadequate or delayed
management.
Objectives: To review the epidemiology and clinical
characteristics of TI in patients (pts) in the US.
Methods: Medical records of pts with a TI diagnosis
(8 mean packed red blood cell transfusions (tx) per
year (yr) over a 3-yr period) and 12 months of fol-
low-up from 1/1997- 4/2014 at 4 US hematology cen-
ters were reviewed for demographic and clinical
characteristics by TI subtype.
Results: Of 138 pts reviewed, 84 had -thal, 39 had -
thal, and 15 had E/-thal. 74% of -thal pts had
deletional (del) Hb H, and 26% had non-del (ndel)
Hb H. Median age was 2.3 yr (1.64 del; 6.1 ndel) in
the -thal group, 9.2 yr in the -thal group, and 2.2 yr
in the E/-thal group. Most -thal (77%) and E/-thal
(87%) pts were Asian, and -thal pts were White
(46%) or African-American (AA; 36%). 21%, 10%,
and 20% of , , and E/-thal pts were foreign-born,
and 5%, 3% and 7% were transfused outside of the
US.
Top 5 comorbidities were splenomegaly,
extramedullary hematopoiesis, growth retardation, he-
patomegaly, and infections needing hospitalization/IV
antibiotics. 22% of pts had 1 tx, while 7% of pts had
Pharmacoepidemiology and Drug Safety, 2016; 25(Suppl. 3): 3681
DOI: 10.1002/pds