12th Congress of the European Hematology ... - Haematologica

12 th Congress of the European Hematology Association
Health economics
0596
COMPLIANCE WITH HOME TREATMENT USING ENZYME REPLACEMENT THERAPY FOR
TYPE 1 GAUCHER DISEASE A UK CENTRES PERSPECTIVE
A. Milligan, R. Bruce, C. Freud, S. Goodwin, D. Hughes, A. Jennings,
L. Richfield, D. Webster, A. Mehta
Royal Free Hospital, LONDON, United Kingdom
Background. Gaucher disease is an inherited metabolic disorder in
which accumulation of glucocerebroside in the reticulo-endothelial system
results in pathology within the bone marrow, liver, spleen and skeleton.
In neuronopathic forms (types 2 and 3) there is CNS involvement.
Treatment with enzyme replacement therapy (ERT) is currently longterm,
possibly life-long. Objective. Management of Gaucher disease
involves the meeting of therapeutic goals. 1 These are: the establishment
and maintenance of specific haematological parameters, reduction in
hepato-splenomegaly, avoidance of bone crises and improvement in
quality of life. Currently the annual drug cost of treating a 70-kg adult
at the recommended dose of 60 iu/kg 2-weekly would be £325,000. UK
practice has been to individualise dose according to clinical status, once
disease has been debulked - making the average cost approximately
£86,000 per patient. 2 Hollak et al. 3 have previously demonstrated the
efficacy of dose titration on goals and disease biomarkers. In the UK
home treatment with ERT for Gaucher was established within 3 years
of the available therapy being licensed and has become the standard.
More than 50% of patients become self-infusers with support from
treating centres and a designated home nursing team. Methods. In view
of this independence we examined compliance amongst our co-hort of
type 1 Gaucher patients receiving treatment with Cerezyme. We used
a patient-led assessment tool and 3 to 6- monthly monitoring with an
approved bio-marker (chitotriosidase). We also compared data to a similar
study on patients having intravenous immunotherapy in the home.
Results. Of 35 patients 21 (60%) stated that they had never missed a
dose of their treatment. 14 (40%) had missed a dose at some time and
the main reason given for this were vacations lasting more than 2 weeks.
Patients were asked to score themselves on a scale of 1 to 10 (1 = noncompliant,
10 = totally compliant, receiving every dose of treatment at
the recommended 2- week intervals). 32 (91%) of patients scored themselves
between 8 and 9 for compliance. 18 patients (51%) had never
experienced any problems with home infusions. Where problems did
occur - 11 (35%) had found some difficulties with cannulation when
they were still learning. 19 (54%) found that home infusion meant that
they felt more in control of their treatment and condition. Summary. In
the UK the treatment of type 1 Gaucher disease with ERT has shown
major clinical benefits to patients. Providing delivery of care in the home
has been beneficial to patients in terms of quality of life and maintenance
of independence. Compliance with unsupervised treatment has not been
shown to be major issue - having positive implications for disease management
in the long-term.
References
1. Pastores, Aerts et al (Therapeutic goals in the treatment of Gaucher disease.
Semin Haematol 2004:41 (4 suppl 5): 4-14).
2. Cannock and Moore (Health Technology Assessment 2006; Vol 10: No.
2.
3. Hollak et al. Blood 2007:109: 387.
0597
CAN WRITTEN ADVICE PROVIDE A SAFE AND ACCEPTABLE ALTERNATIVE TO A NEW
PATIENT ASSESSMENT FOR SELECTED REFERRALS TO HAEMATOLOGY
P. Ganly, 1 H. Keeman, 2 M. Smith, 1 W.N. Patton, 1 R.L. Spearing, 1
S. Gibbons1 1 Christchurch Hospital, CHRISTCHURCH; 2 Christchurch School of Medicine,
CHRISTCHURCH, New Zealand
Background. Traditionally we have seen all patients referred with
haematological problems. An alternative approach for selected patients,
in which the possibility of significant pathology is low, is to provide
written advice to the referrer instead of seeing the patient in person. If
this approach is safe and acceptable to patients and referring doctors, it
may allow resources to be used more effectively for those other patients
222 | haematologica/the hematology journal | 2007; 92(s1)
who require most attention. Aims. We wished to discover what happens
to patients referred and given written advice but not seen and compare
this with other patients who were referred and seen in person. Methods.
After obtaining approval from our IRB we reviewed all referrals to the
department between 11/03 and 06/06. Those referrals which were managed
with written advice (WA) were examined for content of advice
and subsequent course. The diagnoses of these patients were compared
with those referrals subsequently seen non-urgently as new patients
(NUNP). We asked those who referred patients and received written
advice, and recent patients who were managed in this way, for their
opinions on usefulness and acceptability of this advice. Results. We gave
immediate written advice to the referring doctors of 714 (37%) of 1907
referrals. All the remaining referrals were seen according to clinical need,
such that 438 (37%) were seen non-urgently after a wait of 4 weeks or
more (median wait 56 days). All referrals were followed up for a minimum
of 8 months. Diagnoses in WA and NUNP were similar with no
acute pathology in either group. There was a higher proportion of
MGUS, haemochromatosis and haemostasis problems amongst WA and
more myeloproliferative and lymphoproliferative disease amongst
NUNP. 4% of WA v 9% of NUNP have died. 13% of WA were subsequently
re-referred and this led to 52 (7%) attending in person after a
median interval of 4 months from first referral. 21 of these remain current
patients of whom 13 have required treatment. There were 5 patients
who received a diagnosis which had not been predicted at the time of
the written advice letter, and all had been re-referred promptly. 223
(74% of all referring doctors) gave their opinion on this process by questionnaire.
Most were unsurprised by receiving written advice for their
patient(s). 90-98% of respondents believed the process was rapid, helpful,
comprehensive and effective and that they could easily re-refer
patients if necessary. 90% of additional comments, when made, were
favourable. 31 (28% of all patients referred in 2006 whose doctors
received WA) gave their opinion, most being aware of their referral and
pleased not to be seen in the clinic. Most of additional comments, when
made, were favourable. Summary and Conclusions. Providing written
advice as opposed to arranging a new patient visit does not disadvantage
selected patients or miss important pathology. 662 (35% of all referrals)
were not seen at all in the course of our review which allowed
some reallocation of resources to other patients. Skilled referrers, appropriately
supported by written advice overwhelmingly approved of the
process.
0598
COST UTILITY ANALYSIS OF DEFERASIROX VERSUS DEFEROXAMINE (DESFERAL) FOR
PATIENTS REQUIRING IRON CHELATION THERAPY IN THE UNITED KINGDOM
J. Karnon, 1 R.L. Akehurst, 1 K. Jewitt, 2 D. Ossa2 1 2 University of Sheffield, SHEFFIELD; Novartis Plc, CAMBERLEY, United
Kingdom
Background. Patients suffering from β-thalassemia (β-thal), sickle cell
disease (SCD), and myelodysplastic syndrome (MDS) may require lifelong
blood transfusions and are at risk of iron overload. If they are not
treated with iron chelation therapy (ICT), they can suffer serious organ
damage and reduced life expectancy. Desferal, infused subcutaneously
for 8 to 12 hours per day, 5 to 7 times per week, has been the gold standard
of ICT. Exjade is a once daily oral iron chelator, which has been
approved in Europe and the US for the treatment of transfusional iron
overload. Aims. To estimate the incremental cost per quality adjusted life
year (QALY) of using Exjade instead of Desferal in patients with β-thal,
SCD, or MDS who require iron chelation, from a UK NHS perspective.
Methods. A cost utility analysis was performed to compare Exjade with
Desferal. The results from Study 107, a pivotal, open-label, randomised,
controlled study comparing the efficacy of Exjade to Desferal in 586
adults and children with β-thal, demonstrate non-inferiority of Exjade
compared to Desferal in patients who received ≥20 mg/kg/day Exjade.
The cost-utility analysis is therefore based on a comparison of the health
related quality of life associated with the two forms of administration
ie oral therapy versus slow subcutaneous infusion, assuming equivalent
clinical efficacy. Drug costs were informed by observed use in the equivalence
arms of the 107 trial (the ≥20 mg/kg Exjade, and ≥35 mg/kg Desferal
arms)with a mean patient weight of 42kg. The annual equipment
cost for the administration of Desferal was informed by a primary study
of 29 patients (11 β-thal; 14 SCD; 4 MDS; 31% male; mean age
30.6±20.1 years) from four UK treatment centers on the basis of chart
reviews and interviews. The model also includes additional costs associated
with Exjade treatment for creatinine monitoring and adverse
events. A utility study involving 120 UK general population respondents
used the time trade-off approach to estimate utility values for clinically