Abstract
Abstract 4368
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired bone marrow disorder characterised by intravascular hemolysis with resultant anemia often leading to transfusion dependence, severe disabling symptoms and, frequently, life threatening thrombosis. Historically the management of PNH was largely supportive, relatively ineffective and resulted in frequent visits to hospital, admissions, an inability to function normally including loss of employment or other daily activities. PNH is a chronic condition and in most patients persists for the remainder of the patient’s life. Eculizumab was first used for PNH in 2002 and has been licensed since 2007. Eculizumab has been reported to improve all symptoms due to hemolysis in PNH as well as preventing the common complications, such as thrombosis and renal failure and normalising survival. However eculizumab has to be given as an intravenous infusion every 2 weeks indefinitely. In the UK PNH is managed in a shared care model between local hematologists and the National PNH Service from two Centres based in St James’s University Hospital, Leeds and Kings College Hospital, London. Here we report the management of patients treated with eculizumab within the PNH National Service.
A total of 130 patients have been treated with eculizumab since May 2002 with 120 currently receiving therapy. 5 patients have died and none were directly related to PNH or eculizumab. 99 patients requiring transfusions prior to eculizumab have been on treatment for at least a year and 65 (66%) of these have not required transfusions for at least the last 12 months. The rarity of PNH means that patients frequently have to travel long distances for review and treatment. This leads to major issues both in terms of time commitment and expense. In order to allow patients to lead as normal lives as possible we have developed a service model in which Specialist PNH Clinics are performed regionally by the PNH Centre and in which patients receive eculizumab every 2 weeks in their homes delivered by a homecare nursing team. In the UK, the PNH Service and Healthcare at Home Ltd (www.hah.co.uk) have been working in partnership for over 7 years during the clinical trials of eculizumab and since its license in 2007. The PNH Service manages the prescription and delivery of eculizumab including an education program for the homecare nurses. This innovative home infusion programme ensures the safe administration of eculizumab outside of the hospital environment, leading to enhanced treatment-associated convenience for patients and their families. Each year the home infusion program has grown, now over 3000 infusions are given annually including whilst patients are on holiday, visiting family, at University or in the workplace.
A recent patient survey has been conducted from the 2 PNH Centres to assess the patients’ experience of their PNH diagnosis and treatment. 122 patients responded with 70 of these patients receiving treatment with eculizumab and all currently on the home infusion programme. 63 of 68 patients reported the homecare service as excellent or very good compared to 1 reporting it as poor and 66 of the 68 patients preferred to have their treatment at home compared to hospital. The patients main concerns before starting treatment were reduced life expectancy and the requirement for blood transfusions along with fatigue. With eculizumab treatment and the convenience of homecare 30 patients reported being able to return to work. The homecare service is supported by contact between the clinic appointments, 56 of 67 patients having contact with their PNH Specialist Centre by phone or email in addition to the care of the patient’s local hematology team that over 90% of the patients continue to see. The impact of PNH on patients lives before eculizumab treatment was rated and improved from a median of 3 out of 10 (0 = no quality of life; 10 = normal) prior to eculizumab to a median of 8 out of 10 on treatment.
In summary, a novel model of provision of care in PNH with Outreach Specialist Clinics, a 24 hour on call service and homecare delivery of eculizumab permits the normalisation of patients’ lives and overcomes most of the hurdles associated with prolonged regular intravenous therapy. This allows patients to benefit fully from eculizumab including reduction in transfusions, the prevention of serious complications, normalisation of quality of life and where appropriate a return to work.
Disclosures:
Arnold: Alexion Pharmaceuticals: Honoraria. Kelly:Alexion Pharmaceuticals: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau. Hill:Alexion Pharmaceuticals: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau. Richards:Alexion Pharmaceuticals: Honoraria, Speakers Bureau. Elebute:Alexion Pharmaceuticals: Honoraria, Membership on an entity’s Board of Directors or advisory committees. Hillmen:Alexion Pharmaceuticals: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau.
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