Will recent innovations in therapy save perceived deficiencies in self-sufficiency policies.
Future treatment for patients with haemophilia may include the use of either gene therapy, recombinant factor VIII, recombinant factor IX, or high-purity factor IX. Studies on gene therapy are still at the pre-clinical stage, while clinical trials of recombinant factor IX are expected to start by mid-1995. High-purity factor IX concentrates are available and are the treatment of choice for patients with haemophilia B in the absence of a source of recombinant factor IX. Recombinant factor VIII provides a renewable and unlimited source of factor VIII, and is a safe and effective treatment for haemophilia A.
Limentani
S A
SA
Hematology-Oncology Department, New England Medical Center, Boston, MA 02111, USA.
D002986:Clinical Trials as Topic; D017046:Cost Savings; D005164:Factor IX / Q000191:economics / Q000235:genetics / Q000302:isolation & purification / Q000600:supply & distribution* / Q000627:therapeutic use; D005169:Factor VIII / Q000191:economics / Q000235:genetics / Q000600:supply & distribution* / Q000627:therapeutic use; D005171:Factor X Deficiency / Q000191:economics / Q000628:therapy; D005544:Forecasting; D015316:Genetic Therapy; D006467:Hemophilia A / Q000191:economics / Q000628:therapy; D006801:Humans; D011994:Recombinant Proteins / Q000191:economics / Q000600:supply & distribution* / Q000627:therapeutic use; D012449:Safety
D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX