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