Osler-Weber-Rendu Disease (Hereditary Hemorrhagic Telangiectasia) Medication

Updated: Mar 26, 2018
  • Author: Klaus-Dieter Lessnau, MD, FCCP; Chief Editor: Vincent Lopez Rowe, MD  more...
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Medication

Medication Summary

Mild forms of Osler-Weber-Rendu disease (OWRD), also referred to as hereditary hemorrhagic telangiectasia (HHT), do not require treatment. When treatment is indicated, agents to be considered include iron salts, estrogen-based formulations, antifibrinolytics, and immunomodulators.

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Iron Salts

Class Summary

Iron replacement therapy provides symptomatic relief in patients with anemia.

Ferrous sulfate (Feosol, Fer-In-Sol, Fer-Iron, Iron Supplement Children's)

This is the mainstay of treatment for patients with iron deficiency anemia. It should be continued for about 2 months after correction of the anemia and its etiologic cause in order to replenish body stores of iron. Ferrous sulfate is the most common and cheapest form of iron used. Tablets contain 50-60 mg of iron salt. Other ferrous salts are used and may cause less intestinal discomfort because they contain a smaller dose of iron (25-50 mg). Oral solutions of ferrous iron salts are available for use in pediatric populations.

Iron dextran (Dexferrum, INFeD)

Iron dextran is used to treat microcytic, hypochromic anemia resulting from iron deficiency. It replenishes depleted iron stores in the bone marrow, where it is incorporated into hemoglobin. Parenteral use of iron-carbohydrate complexes has caused anaphylactic reactions, and its use should be restricted to patients with an established diagnosis of iron deficiency anemia whose anemia is not corrected with oral therapy. The required dose can be calculated (3.5 mg iron/g of hemoglobin) or obtained from tables in the Physician's Desk Reference (PDR). For intravenous use, INFeD may be diluted in 0.9% sterile saline. Do not add it to solutions containing medications or parenteral nutrition solutions. A test dose of 0.5 mL (in children, 0.25 mL) should be administered before therapy is started. This agent is available as 50 mg iron/mL (as dextran).

Iron sucrose (Venofer)

Iron sucrose is used to treat iron deficiency in patients in whom absorption of iron from the gastrointestinal (GI) tract is insufficient. The incidence of anaphylaxis is lower with iron sucrose than with other parenteral iron products.

Ferric gluconate (Ferrlecit)

Ferric gluconate replaces the iron found in hemoglobin, myoglobin, and specific enzyme systems, allowing transportation of oxygen via hemoglobin.

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Estrogens/Progestins

Class Summary

Oral contraceptives may be used to reduce bleeding. Topical preparations may be used to help strengthen the mucosa and decrease the susceptibility of the mucosa to external trauma. Before use, screening tests for pulmonary arteriovenous malformations (AVMs) should be performed because of the risk of complications involving thromboembolism.

Estrogen therapy may also be beneficial in some women with HHT and may be used to decrease the amount of bleeding. Oral contraceptives have been shown to be more effective than estrogen alone in mucosal bleeding.

Norethindrone acetate and ethinyl estradiol (Junel 1.5/30, Microgestin 1.5/30, Loestrin 21 1.5/30)

Norethindrone acetate−ethinyl estradiol is used to decrease mucosal bleeding. It probably works by strengthening mucosal tissues and thereby making them more resistant to trauma.

Estradiol (Estrace, Climara, Femring, Elestrin, Alora, Minivelle, Vivelle-Dot)

Estradiol increases synthesis of DNA, RNA, and many proteins in target tissues. Norethindrone acetate−ethinyl estradiol is an option.

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Antifibrinolytic Agents

Class Summary

Antifibrinolytic agents are used to enhance hemostasis when fibrinolysis contributes to bleeding. In view of the risk of thromboembolic events, screening for pulmonary AVMs should be performed before these agents are used.

Aminocaproic acid (Amicar)

Aminocaproic acid inhibits fibrinolysis through inhibition of plasminogen activator substances and, to a lesser degree, through antiplasmin activity. It is used to prevent or treat mucosal bleeding caused by bleeding disorders or trauma.

Tranexamic acid (Cyklokapron)

Tranexamic acid is an alternative to aminocaproic acid. It inhibits fibrinolysis by inhibiting plasminogen activators.

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Immunomodulators

Class Summary

Case reports have documented regression of OWRD lesions with the use of interferon alfa in patients who were treated for other indications.

Interferon alfa-2a (Pegasys)

Interferon alfa-2a is a protein product manufactured by recombinant DNA technology. The mechanism of its antitumor activity is not clearly understood; however, direct antiproliferative effects against malignant cells and modulation of host immune response may play important roles.

Peginterferon alfa-2b (PEG-Intron)

PEG-IFN consists of interferon alfa-2b attached to a single 12-kd polyethylene glycol (PEG) chain. It is excreted by the kidneys. PEG-IFN has sustained absorption, a slower rate of clearance, and a longer half-life than unmodified interferon, which permits more convenient once-weekly dosing and significantly improves quality of life for patients.

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Antineoplastics, Monoclonal Antibody

Class Summary

These agents may inhibit microvascular growth, which, in turn, may retard the growth of all tissues, including metastatic neoplastic growth.

Bevacizumab (Avastin)

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