Congential Hemophilia A

Updated: May 29, 2023

Background

Epidemiology

Anatomy

Pathophysiology

Etiology

Genetics

Prognostic Factors

Clinical History

Physical Examination

Age group

Associated comorbidity

Associated activity

Acuity of presentation

Differential Diagnoses

Laboratory Studies

Imaging Studies

Procedures

Histologic Findings

Staging

Treatment Paradigm

by Stage

by Modality

Chemotherapy

Radiation Therapy

Surgical Interventions

Hormone Therapy

Immunotherapy

Hyperthermia

Photodynamic Therapy

Stem Cell Transplant

Targeted Therapy

Palliative Care

Medication

valoctocogene roxaparvovec-rvox

Gene therapy using an adeno-associated virus vector is authorized by the FDA for the treatment of severe hemophilia A (congenital factor VIII insufficiency with factor VIII activity 1 IU/dL) in individuals who have never been exposed to the virus and do not have antibodies against adenovirus serotype 5.

Administer 6 x 1013 vector genomes/kg as a one-time intravenous injection.
Calculating the dosage in milliliters (mL) and the number of vials necessary
Patient dosage volume in milliliters: Dose in mL equals body weight in kg x 3
The per kilogram dosage (6 x 1013 vg/kg) is represented by multiplication factor 3, divided by the suspension's vg/mL concentration (2 1013 vg/mL).
Vials that need to be thawed: The number of vials that need to be thawed equals the patient dosage volume in milliliters divided by eight, rounded up to the nearest full vial.
The minimum volume that may be extracted from a vial is represented by division factor 8 (8 mL).

fluprednisolone

To suppress inhibitors in haemophilia A using continued treatment with factor VIII, administer dose of 0.5 mg/kg of body weight for 3 weeks until reaching correct in vivo recovery after therapeutic administration

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