Page 29 - Roche Hemlibra Non-inhibitors - Product Monograph

POOLED DATA FROM FOUR HAVEN STUDIES

• No deaths, thrombotic, or thrombotic microangiopathy events were observed beyond those
reported in the HAVEN 1 primary analysis (Oldenburg et al. 2017)
• 103 SAEs were reported in 71 participants (Callaghan M et al. 2019)

o serious adverse event (SAEs) reported by ≥5 participants were haemorrhage (n = 7, 1.8%)
and hemarthrosis (n = 5, 1.3%)

• The most common treatment-related AEs were injection-site reaction† (n = 104; 26.1%)
(Callaghan M et al. 2019)

• ADAs with neutralizing potential were observed in <1% (3/398) of participants (Callaghan M et al.
2019)

Long-term safety
Total (N = 399) *

Total number of participants with ≥1 AE, n (%) 373 (93.5)

Total number of patients, n (%) 1 (0.3)
AE with fatal outcome 71 (17.8)
Serious AE 5 (1.3)
AE leading to withdrawal from treatment 73 (18.3)
Grade ≥3 AE 134 (33.6)
Related AE 107 (26.8)
Local injection-site reaction†

Adverse events of special interest 1 (0.3) ‡
Systemic hypersensitivity/anaphylactic/anaphylactoid reaction 3 (0.8)
TMA event related to concomitant aPCC and emicizumab 2 (0.5)
TE related to concomitant aPCC and emicizumab 1 (0.3)
Other TE (grade 1 device occlusion)

* The safety population only included those patients who received emicizumab. One participant in HAVEN 1 discontinued prior to
emicizumab treatment and was excluded from the safety analyses.
† All ISRs were mild in severity. ‡Assessed using the Sampson Criteria and including all participants that experienced indicative
symptoms.
7.4. QUALITY OF LIFE (QoL): HAVEN 3 AND 4

PROPHYLAXIS VS EPISODIC THERAPY IN HEMOPHILIA (WHF guidelines. 2020)
The updated results of HAVEN 3 and HAVEN 4 came from the assessment of impact of
prophylactic emicizumab on HRQoL of PwHA with/without FVIII inhibitors.

HRQoL was assessed by the Haem A QoL questionnaire, with 143 and 38 eligible respondents
(aged ≥18 years) for HAVEN 3 and 4, respectively.

Methodology

• HRQoL data were pooled by study regardless of patient baseline characteristics or treatment

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