This document discusses a study examining the needs and challenges of people with hemophilia A in North Macedonia. The study found that over half of the surveyed participants had severe hemophilia A and were treated with plasma-derived factor VIII on an on-demand basis. Most participants reported joint complications from repeated bleeding episodes. Participants expressed interest in novel therapies, especially monoclonal antibodies, that could enable home-based prophylactic treatment and reduce joint damage and impact on quality of life. The study highlights the need to overcome treatment challenges through alternative therapies for people with hemophilia A in North Macedonia.

1. UNDERSTANDING THE NEEDS OF PEOPLE WITH HEMOPHILIA A
WILL ENHANCE TO OVERCOME THE CHALLENGES IN NORTH MACEDONIA
Dejanova-Ilijevska V.,1 Makarovska-Bojadjieva T.,1 Velkova E.,1 Grubovic-Rastvorceva R.,1 Petkovic E.,1 Bogdanovska-Ristovska E.,1 Ziberi M.,2
Nakjeska C.,2 Nakjeska M.,2 Kocev I.,3 Lulevik D.,3 Smokovski A.4
1 Institute for Transfusion Medicine, Skopje, North Macedonia 2 Bleeding Disorders Society HEMOLOG Macedonia 3 Civil Society with coagulopathies HEMO-WILL, Ilinden
4 Roche Macedonia DOOEL Skopje, North Macedonia
Presented at the 5th Congress of Macedonian Society for Transfusion Medicine with international participants; October 16-19 2019; Skopje, Republic of North Macedonia
BACKGROUNDS
AIMS
METHODS
RESULTS
REFERENCES
1. WFH. Guidelines for the management of hemophilia. 2012. Last accessed 08 June 2016
http://www1.wfh.org/publications/files/pdf-1472.pdf. 2. WFH. Guidelines for the
management of hemophilia. 2012. http://www1.wfh.org/publications/files/pdf-1472.pdf.
3.Berntorp E, Shapiro AD. Modern haemophilia care. The Lancet 2012; 370:1447-1456. 4.
Elder-Lissai A, Hou Q, Krishnan S. The Changing Costs of Caring for Hemophilia Patients in
the U.S.: Insurers’ and Patients’ Perspectives. Presented at: ASH December 6-9, 2014.
Abstract # 199.
Hemophilia is a serious, inherited bleeding disorder leading
to severe cases to uncontrolled bleeding, either
spontaneously or after minor trauma. Recurrent bleeds can
lead to significant impairment, especially in their joints.
Hemophilia A is the most common type of haemophilia1,
affecting approximately 320,000 people around the
world.3,4 Current treatment is replacement of FVIII,
recombinant (r) or plasma derived (pd), on-demand or
prophylactically. But frequent intravenous (IV) infusions are
burden for most people with hemophilia A (PwHA) and
their caregivers.4
The objective was to examine the current medical needs
and challenges that people with hemophilia are facing in
North Macedonia.
A standard questionnaire was created only for this study
and survey was initiated by the three main Hemophilia
Patient Associations in Republic of North Macedonia,
supported by Roche Macedonia. The survey was conducted
during September 2019. Each participant signed consent
form prior participation.
In total 23 persons participated, from whom 18 (78.3%)
were males with hemophilia A, the rest were caregivers
who answered the questions for their sons. According to
the participants, in 47.8% the disease was severe, severe
to moderate was in 21.7%, moderate in 26.1% and mild in
4.1% (Figure 1). The median age was 39.5 years (range 16-
62) and no one had FVIII inhibitors. 52.2% of surveyed
PwHA were treated with pdFVIII, 39.1% with rFVIII and
8.7% didn’t know what kind of treatment they used last.
CONCLUSIONS
 Our study revealed that more than half of the
surveyed PwHA were on on-demand IV treatment
with pdFVIII, and were significantly impacted by
joint complications.
 Introduction of novel therapies as home prophylaxis
might potentially overcome those challenges and
increase the PwHA quality of life.
Figure 2. Preference of treatment type
47,8%
21,7%
26,1%
4,1%
severe severe to moderate moderate mild
Figure 1. Disease severity according to the participants
On-demand treatment was used in 64.2%, the rest were
on prophylaxis. 52.2% stated that they would like to use
monoclonal antibody, 17.4% rFVIII, 17.4% pdFVIII and
gene therapy only 13% (Figure 2).
13% accepted up to 12 per year (Figure 3). Majority, 87% of
PwHA, had joint complications as a result of repeated
bleedings and in 70% those damages had significant
impact.
n = 23
52,2%
17,4%
26,1%
13%
monoclonal antibody rFVIII pdFVIII gene therapy
56.5% from all would like to be treated prophylactically,
43.5% on-demand, where by 60.9% would like to be
treated at home. 69.6% agreed that IV administration is
long, complicated and causing discomfort, 17.4% were
neutral and the rest didn’t agree with this. More than
half (56.5%) answered that zero bleeds are acceptable,
17.4% >3 bleeds per year, 13% from 3-6 bleeds per year,
Hemophilia A affected the quality of life in 95.7%, only
4.3% were not affected. In general, 82.6% were satisfied
beside only 17.4% who were unsatisfied from the
healthcare provided.
n = 23
56,5%
17,4%
13%
13%
zero >3 3-6 12
Figure 3. Acceptable bleeds per year