Thrombasthenia Treatment & Management

Updated: Oct 25, 2018
  • Author: Vivian Y Chang, MD, MS; Chief Editor: Robert J Arceci, MD, PhD  more...
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Treatment

Medical Care

Refractory bleeding in individuals with thrombasthenia requires the transfusion of normal platelets. Use human leukocyte antigen (HLA)–matched platelets whenever possible to prevent alloimmunization complications.

Epistaxis can be controlled with nasal packing or application of gel foam soaked in topical thrombin. Case reports have described nasal packing with cured salted pork in the form of a nasal tampon being an effective therapy for uncontrollable epistaxis in a patient with Glanzmann thrombasthenia (GT), [15] as well as use of the medicinal plant extract Ankaferd Blood Stopper for gingival bleeding. [16] E-aminocaproic acid may be used to control bleeding in patients with severe epistaxis or after dental extraction.

Menorrhagia can be severe in patients affected with GT. It usually results from excessive estrogen secretion, which causes a highly proliferative endometrium. Acute menorrhagia can be treated with high doses of progesterone followed by maintenance therapy with oral contraceptive pills.

Iron deficiency anemia can develop in patients with chronic gingival bleeding, GI bleeding, or menorrhagia and may require oral iron supplementation.

Recombinant factor VIIa (rFVIIa) has been successfully used to achieve hemostasis in patients with GT, [17] particularly in patients who have developed isoantibodies to the GP IIb-IIIa complex and who are thus refractory to platelet transfusions. [18] rFVIIa appears to enhance endothelial deposition of GP IIb-IIIa–deficient platelets. Combined with antifibrinolytic therapy, rFVIIa further stabilizes the newly formed clot. [7] rFVIIa has been approved by the European Union for use in patients with GT and platelet refractoriness due to antibodies.

The use of rFVIIa continues to be investigated in the setting of GT. It is currently not approved by the US Food and Drug Administration (FDA) for patients with GT, and its use in children with thrombasthenia remains controversial. It should be noted that off-label use of rFVIIa has not been shown to provide an overall mortality benefit and may increase the risk of thromboembolism. [19, 20, 21, 22] Optimal dosing has yet to be defined in both pediatric and adult patients with GT. A dose of 90 mcg/kg given every 2 hours for 3 or more doses has been used with success. [18]

Antihemophilic factor VIII/von Willebrand factor concentrate (Humate-P) has been used in active bleeding, refractory to rFVIIa. [23] The von Willebrand factor component may increase platelet plug formation. However, pre-Humate-P and post-Humate-P laboratory testing of platelet aggregation studies, as well as thrombin levels and activity, were not significantly affected.

HLA-matched sibling allogeneic stem cell transplantation (SCT) has been successfully performed in patients with GT and platelet isoantibodies that cause severe refractory bleeding. Although curative, this treatment is not recommended in routine cases of thrombasthenia because of the potential complications associated with SCT. The decision to pursue SCT as a potential treatment for GT should be made on a case-by-case basis. Reduced intensity conditioning regimens with SCT have been used in small series with excellent results. [24] This raises the possibility of curative therapy with reduced adverse late effects. Most recently, patients with GT who have undergone successful unrelated donor cord blood transplantations have been reported; this may be an option for patients without matched-sibling donors. [25, 26]

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Surgical Care

Treatment with platelet transfusions is often necessary prior to surgical or dental procedures. Aminocaproic acid may also be used postsurgically to control bleeding. Recombinant factor VIIa has been used in invasive surgical procedures or high-risk surgical procedures such as cardiothoracic surgery and spinal/neurosurgery. For dental extractions, molded plastic splints can aid in achieving hemostasis.

Pregnancy and especially delivery can be a major challenge for patients with GT and their care providers. HLA-matched platelet transfusions should be given prior to delivery and are usually required for a week postpartum. [7] rFVIIa has been used successfully to treat severe bleeding at delivery.

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Consultations

Consultation with a hematologist is strongly suggested.

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Diet

No special diet is required. However, patients with GT should avoid consuming excessive quantities of foods and substances that further interfere with platelet function. This includes excessive quantities of garlic, onions, ginger, and ginseng, as well as food products with quinine and aspirin.

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Activity

Any degree of trauma in a patient with thrombasthenia can be severe.

Advise persons with thrombasthenia to take appropriate limitations and precautions with sports and other activities.

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