Abstract Number: PB0712
Meeting: ISTH 2021 Congress
Background: Hereditary factor XIII deficiency (FXIIID) is a rare recessive autosomal disease. Accurate diagnosis and early start of FXIII prophylaxis are key to prevent abnormal bleedings.
Aims: To describe the effectiveness and the safety of FXIII prophylaxis, and the health-related quality of life (EuroQol-5 Dimension or EQ-5D) of people with FXIIID (PwFXIIID) on prophylaxis treated at HEMOMINAS, Belo Horizonte/Brazil.
Methods: PwFXIIID on FXIII prophylaxis were invited. Clinical data were reviewed using a standardized file. Subjects self-administered the EQ-5D, at study entry.
Results: Of the 11 PwFXIIID registered, 9 (82%) signed the Consent Form (Table 1). Prophylactic regimens ranged from 9.1 to 18.8 IU/kg monthly. Since the start of prophylaxis, 5/9 and 3/9 patients reported spontaneous or post-traumatic bleeding which required FXIII infusion, respectively. No arterial/venous thrombotic events were reported during the prophylaxis period.
|Age at study entry (year)||43||23||23||19||50||14||28||27||30|
|Age at diagnosis (year)||32||4||2||7||48||13||6||1||2|
|Age at start of prophylaxis (year)||33||20||2||16||48||13||23||3||15|
|Monthly dose [IU (IU/kg)]||750 (13.2)||1000 (15.6)||750 (9.1)||750 (12.6)||1500 (18.8)||750 (11.5)||750 (10.1)||1000 (12.3)||1000 (11.5)|
|Treated spontaneous bleedings during prophyaxis||3||1||4||0||4||0||2||0||0|
|Treated post-traumatic bleedings during prophylaxis||1||0||0||0||0||2||1||0||0|
Only 3/9 patients reported both “no problems” in the 5 dimensions of EQ-5D and high levels of QoL in the EQ-VAS (Table 2). One patient reported “some problem” or “extreme problem” in all EQ-5D dimensions, and a low level of EQ-VAS. While “self-care” was usually considered as “no problem” by most patients (8/9), “pain/discomfort” and “anxiety/depression” were reported as problematic by almost half of them.
|Quality of life \ Patient#||#1||#2||#3||#4||#5||#6||#7||#8||#9|
|Mobility||some problem||no problem||no problem||no problem||no problem||no problem||no problem||no problem||some problem|
|Self-care||some problem||no problem||no problem||no problem||no problem||no problem||no problem||no problem||no problem|
|Usual activities||some problem||no problem||some problem||no problem||no problem||no problem||no problem||no problem||some problem|
|Pain/disconfort||extreme problem||no problem||some problem||some problem||no problem||no problem||no problem||no problem||some problem|
|Anxiety/depression||extreme problem||no problem||some problem||no problem||some problem||extreme problem||no problem||no problem||no problem|
Conclusions: To our knowledge, this is the first report about EQ-5D evaluation among PwFXIIID on prophylaxis, which is the standard treatment with a good safety profile. In general, QoL was well perceived, although some patients reported severe symptoms in few or all domains. Unfortunately, we could not find a cause of this profile, since clinical-outcome characteristics were too heterogenous. We are elaborating future registries involving patients from other Brazilian states, to better clarify both treatment characteristics and QoL of PwFXIIID. We believe QoL should be formally evaluated in PwFXIIID, and the multiprofessional team should try to help him/her to overcome the reported troubles.
To cite this abstract in AMA style:Drumond A, RC. Quality of Life of People with Hereditary Factor XIII Deficiency Treated at a Reference Centre [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/quality-of-life-of-people-with-hereditary-factor-xiii-deficiency-treated-at-a-reference-centre/. Accessed November 27, 2021.
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