Our experience in providing home self-administered therapy to HAE
patients
Maria Bova1*, Angelica Petraroli1, Stefania Loffredo1, Maria Concetta Siani1 and Massimo Triggiani2
1Division of Clinical Immunology and Allergy and Center for Basic and Clinical Immunology Research (CISI),
University of Naples “Federico II”, Naples, Italy; 2Division of Internal Medicine, University of Salerno, ItalyBACKGROUND
Hereditary angioedema (HAE) due to C1-inhibitor deficiency is a rare
autosomal dominant genetic disorder characterized by recurrent
attacks of subcutaneous and/or submucosal edema. Replacement
therapy with plasma-derived C1-inhibitor (pdC1INH) is used to treat
acute HAE attacks. It is administered endovenously. New guidelines
indicate that home pdC1INH self-administration programs should be
offered to patients. We describe our experience in the organization of
self-administration courses offered to HAE patients living in Campania
(Italy).
Table 2: Characteristics of the patients population
Gender (Patients N=30)• Male 19• Female 11
Median age (years) • Patients 25.4 (range 5-
40)• People attending the courses 38.5 (range 17-
50)(including patients’ parents )
Patients on prophilaxis (N)• pdC1INH 2 (range 12-31)• Danazol 5 ( range 25-38)
Table 1: Diagnosis of HAE
Definition of HAE
History of recurrent angioedema without major urticaria
Recurrent abdominal colicky pain attacks
Onset of symptoms before the age of 20 years
Presence of family history of angioedema
Evidence of antigenical and /or functional C1 INH plasma levels below 50%
of normal on 2 separate determinations
Table 3: Impact of home therapy (HT) on patients’ life
Quality of life Improved
Total hospitalization due to HAE (N; median) 11/year 3.5/year
Total number of lost work/school days (N; median) 8/year 5/year
Time to begin pdC1INH infusion (hours; median) 5 2.5
Time to attack resolution (hours; median) 10 8
Before HT After HT
Figure 1: Hereditary angioedema acute attacks
METHODS
60 patients with HAE are seen in our department. Until the past year
they used to treat their acute attacks at the hospital or at home cared
by a healthcare professional (nurse or MD). Since the past year we
offered to the patients the possibility to attend self-administration
courses. We have organized 2 courses until now involving 30 patients
or their relatives (for the children we involved the parents obviously).
The teacher was a nurse with a specific experience in the training in
intravenous self-administration. We indicated to the patients to go to
the nearest hospital in case of laringeal attacks in spite of their ability
to self-administer pdC1INH.
1) Backgroud information on the disease and treatment possibilities
2) Indications for administration of pdC1Inh concentrate (Every patient have agreed
with a healthcare professional on a specific treatment regimen (how often,
how much)
3) Storage, reconstitution and administration of pdC1Inh through venipuncture with a
butterfly needle (until the patient feels to have adequate education on self-
infusion)
4) Correct aseptic techniques and how to use properly needles and syringes
5) Instruction in emergency treatment at the local hospital in case of laringeal attacks or
treatment failure
6) Management of adverse reactions
7) Documentation of each C1Inh concentrate replacement (including dose and batch
number) and swelling attack in a diary with registration of adverse effects
8) Advice as to when to consult a physician if the attack symptoms are atypical
9) Initial monitoring by a healthcare professional of individual's technique
10) Periodic reassessment of educational needs and techniques to perform home
infusionTable 4: Home therapy training programme step by step
RESULTS
The patients attended the courses with attention. Some of them were
able to self-administer pdC1inh immediately. Others had some
difficulties. These patients were offered a home-care by our expert
nurse. After some weeks of home courses all the patients or their
relatives are now able to administer pdC1INH. After some months of
self-administration all patients declare their quality of life is better
than before. They seem to treat the attacks earlier than before with a
consequent faster resolution.
CONCLUSIONS
The majority of patients are capable of conducting home treatment by
self-administration of pdC1INH after a specific training. Home
treatment and self-administration offer a good alternative to the
hospital treatment and seems to improve patients’ life.
Home pdC1Inh infusion should be offered to all the HAE patients
