Preconception or intrapartum counselling and informed consent for female hemophilia A and B carriers
| Study | Summary |
|---|---|
| Hermans et al.,42 2018 | Description of the evolution of practice, and unmet needs and options for girls and women in families with hemophilia, as well as the clinical and laboratory characteristics during pregnancy. |
| Hermans et al.,82 2022 | Choosing the optimal treatment using an integrated, patient-oriented approach to shared decision-making between patients and clinicians. |
| Mannucci,83 2020 | Despite the major advances in prophylaxis obtained with extended half-life factor products and nonfactor therapies, break-through bleeding has not been fully eliminated, and treatment is still physically and psychologically invasive, even when the subcutaneous route of administration is used. |
| Delgado-Flores et al.,84 2022 | Results suggest that prophylactic treatment (at low, intermediate or high dosages) is superior to episodic treatment for bleeding prevention. For patients with hemophilia A, bleeding rates seem to have a dose–response effect. |
| Ellsworth et al.,85 2021 | Review of background, rationale and potential of nonfactor therapies, as well as pitfalls and expected limitations. Discussion of factor mimetic therapy (emicizumab), siRNA therapeutics, tissue factor pathway inhibitors and serine protease targets. |
| Kenet et al.,86 2021 | Despite high adherence to prophylaxis, the continued occurrence of spontaneous and joint bleeding events requiring treatment, and impaired physical functioning was evident in this study. These results illustrate real-world shortcomings associated with regular FVIII prophylaxis for a cohort of patients with severe hemophilia A, for whom additional hemostatic options were needed. |
| Witarto et al.,87 2022 | This analysis indicates that rurioctocog alfa pegol could serve as a safe and effective alternative for bleeding prophylaxis in previously treated patients with hemophilia A; however, there are limited direct comparison studies. Moreover, this therapy appears to have low immunogenicity, which further increases the safety profile of the drug in such clinical conditions. |
| George,88 2021 | Current clinical hemophilia gene therapy efforts are focused largely on the use of systemically administered recombinant adeno-associated viral (rAAA) vectors for FVIII or FIX gene addition. |
| Arruda et al.,89 2021 | Gene therapy for hemophilia A and B has benefited from advancements in the field of general gene therapy, such as the development of adeno-associated viral vectors and disease-specific breakthroughs, such as the identification of B-domain deleted FVIII and hyperactive FIX Padua. It has also benefited from hemophilia B clinical studies that revealed critical safety concerns related to immune responses to the vector capsid not anticipated in preclinical models. |
| Gualtierotti et al.,90 2022 | The management of pain in patients with hemophilia requires more standardization. Pain management is based on analgesics such as paracetamol, the first-line treatment of acute and chronic pain in adults and children, in association with opioids in adults. Since NSAIDs inhibit platelet function, short courses of these drugs are required. Local treatment with intra-articular injections of corticosteroids is an option for refractory cases. Physiotherapy has a role after hemarthrosis and for long-term management of chronic pain for both pediatric and adult patients. |
FIX = factor IX; FVIII = factor VIII; NSAID = nonsteroidal anti-inflammatory drug; siRNA = small interfering RNA.