Novel treatments for epistaxis in hereditary hemorrhagic telangiectasia: a systematic review of the clinical experience with thalidomide

@article{Franchini2013NovelTF,
  title={Novel treatments for epistaxis in hereditary hemorrhagic telangiectasia: a systematic review of the clinical experience with thalidomide},
  author={Massimo Franchini and Francesco Frattini and Silvia Crestani and Carlo Bonfanti},
  journal={Journal of Thrombosis and Thrombolysis},
  year={2013},
  volume={36},
  pages={355-357}
}
Hereditary hemorrhagic telangiectasia (HHT) is a rare, autosomal dominant bleeding disorder that affects one in 5,000–8,000 individuals [1, 2]. It is characterized by mucocutaneous telangiectasia and arteriovenous malformations in the pulmonary, cerebral and hepatic circulations [3]. The disorder is primarily caused by mutations in genes involved in vascular development and repair, e.g. the endoglin gene (ENG) located on chromosome 9 and the gene encoding activin receptor-like kinase-1 (ALK-1… 

Optimal management of hereditary hemorrhagic telangiectasia

An overview of the clinical features of HHT, discusses the clinical and genetic diagnostic strategies, and presents an up-to-date review of literature and detailed considerations regarding screening for visceral AVMs, preventive modalities, and treatment options are presented.

Current and emerging pharmacotherapies for hereditary hemorrhagic telangiectasia

This review includes the own experience of years researching to find therapeutic agents in HHT, and the use of antifibrinolytics, modulators of hormonal receptors (SERMs), anti-oxidant agents, immunosuppresants and anti-angiogenic agents, tested under preclinical conditions.

Thalidomide for Epistaxis in Patients with Hereditary Hemorrhagic Telangiectasia: A Preliminary Study

  • Jia FangXiaomeng Chen K. Su
  • Medicine
    Otolaryngology--head and neck surgery : official journal of American Academy of Otolaryngology-Head and Neck Surgery
  • 2017
Thalidomide may be a treatment choice for recurrent epistaxis in HHT patients, although the side effects should be considered, and guidelines for dosing and course should be focused on.

Anti-angiogenic therapeutic strategies in hereditary hemorrhagic telangiectasia

The experimental evidence for dysregulated angiogenesis in HHT, the anti-angiogenic therapeutic strategies used in animal models and some patients with HHT and the potential benefit of theAnti-Angiogenic treatment for ameliorating this severe, progressive vascular disease are reviewed.

The use of thalidomide therapy for refractory epistaxis in hereditary haemorrhagic telangiectasia: systematic review.

Current available evidence suggests that low-dose thalidomide is effective in transiently reducing epistaxis frequency and duration, and further studies are required to establish a treatment regimen to prevent side effects.

Follow-up of Thalidomide treatment in patients with Hereditary Haemorrhagic Telangiectasia.

Despite symptom reduction, alternative treatments are still necessary for epistaxis in HHT patients and a large-scale clinical trial is not justified although incidental use in the most severely affected patients can be considered.

Hereditary Hemorrhagic Telangiectasia: The ENT point of view

This review focuses on Hemorrhagic Hereditary Telangiectasia, a familiar, evolving and potentially life-threatening disease, whose management can be sometimes a real nightmare for the clinician.

Novel Use of Nasal Septal Button for Epistaxis in HHT Syndrome: An Observational Study

The placement of nasal silicone septal button was shown to incidentally decrease significantly the incidence of bleeding in one patient by isolating the sePTal mucosa and the same procedure was then offered to the two other patients with similar success in decreasing bleeding and transfusion rate over 4 years of follow up.

References

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Efficacy of Thalidomide in the Treatment of Severe Recurrent Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT): Preliminary Results of an Ongoing Study

These preliminary results strongly support the hypothesis that low-dose thalidomide is very effective for the treatment of epistaxis in patients with severe HHT who did not benefit from other available modalities of treatment.

Long‐Term Therapy With Thalidomide in Hereditary Hemorrhagic Telangiectasia: Case Report and Literature Review

A case of HHT complicated with persistent GI bleeding and refractory anemia is reported, where the patient was able to resume daily activities and maintain a stable hematological profile and thalidomide treatment showed improvement in the hepatic telangiectasia.

Hereditary hemorrhagic telangiectasia: from molecular biology to patient care

The goal of this review was to describe the underlying molecular and cellular physiopathology, explore clinical and genetic diagnostic strategies for HHT, and present clinical management recommendations in order to treat symptomatic disease and to screen for vascular malformations.

Hereditary hemorrhagic telangiectasia: An overview of diagnosis, management, and pathogenesis

Molecular genetic testing is used to establish the genetic subtype of hereditary hemorrhagic telangiectasia in a clinically affected individual and family, and for early diagnosis to allow for appropriate screening and preventive treatment.

A review on clinical management and pharmacological therapy on hereditary haemorrhagic telangiectasia (HHT).

A systematic review and the beginning of a systematic laboratory work to screen for "orphan drugs" as therapeutic agents in HHT, where the use of hormones, immunosuppresants and anti-angiogenic agents are under preclinical study in the laboratory.

Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia

The ability of thalidomide to induce vessel maturation, which may be useful as a therapeutic strategy for the treatment of vascular malformations, is demonstrated.

Hereditary haemorrhagic telangiectasia: a clinical and scientific review

The underlying molecular, cellular and circulatory pathobiology of HHT is reviewed; clinical and genetic diagnostic strategies are explored; detailed considerations regarding screening for asymptomatic visceral involvement are presented; and overviews of management strategies are provided.

Using thalidomide in a patient with epithelioid leiomyosarcoma and Osler-Weber-Rendu disease.

Thalidomide (Thalomid) is recognized to have antiangiogenic properties and has been shown to be effective in the treatment of refractory myeloma.[1] As a result, thalidomide is now being investigated

Deep vein thrombosis induced by thalidomide to control epistaxis secondary to hereditary haemorrhagic telangiectasia.

This is the first report of venous thromboembolic complication induced by thalidomide prescribed in this setting and this agent should be used with great caution in this indication, given its thrombogenicity and difficulties to manage systemic anticoagulation in patients with HHT.