Directory of Drugs Therapeutic Area Updates

Hematology

There are currently about 3.5 million people diagnosed with hematologic disorders in the United States. Anemia is the most commonly diagnosed hematologic disorder, with estimates as high as 3.4 million people. Even so, there are indications that millions of others suffer from anemia but are currently undiagnosed. Other significant hematologic disorders include thrombocytopenias, white blood cell disorders (such as neutropenia) and hemophilia.

Current statistics for these conditions are:

Condition

Prevalence

Anemia

3.4 million people

- sickle cell anemia

1 in 400 African Americans

- thalassemia, homozygous

1,000 people

Idiopathic thrombocytopenia purpura (ITP)

100,000 people

Hemophilia

17,000 people

Of additional significance are people who need treatment for blood-related problems that result as a consequence of treatment for another medical condition or of another disorder. For example, anemia secondary to surgical procedures or trauma is common, and white blood cell disorders - such as neutropenia - are a frequent consequence of chemotherapy. Autoimmune disorders often present with low hematocrits and low platelet counts.

Drugs (or pseudo-hemoglobins) that effectively mimic the action of the oxygen-carrying role of hemoglobin can play a very large role in the management of anemic disorders. Furthermore, such drugs have the promise of decreasing the need for blood transfusions in patients undergoing surgery as well as decreasing the mortality rate in major trauma victims.

Some of the drugs under development in this area include a human red blood cell substitute called pyridoxalated hemoglobin polyoxyethylene, Hemopure (a hemoglobin-based oxygen therapy solution originally derived from cows) and Hemolink (a human hemoglobin-based oxygen carrier) for use in coronary artery bypass grafting surgery. These drugs are all in late phase testing at the time of this publication.

Sickle cell anemia is another area with drugs under development. One drug showing potential is Flocor (poloxamer 188 N.F.), which recently completed a phase III trial for sickle cell subjects suffering from vascular occlusive crisis. While the drug was not granted FDA approval, there was enough efficacy in subject subgroups that the recommendation was made to organize another phase III efficacy study.

Other areas of intense investigation recently include the treatment of thrombocytopenia (Argatroban (Texas Biotechnology Corporation) was approved for the treatment of heparin-induced thrombocytopenia) as well as the treatment of hemophilia. Many of the gains in the area of hematology are being expanded to related fields. For instance, Neumega (Oprelvekin) (Genetics Institute, Inc.) is being used to treat chemotherapy-induced thrombocytopenia; however, phase II/III trials are under way currently for the use of Neumega in Crohn's disease. Similarly, Colirest (CBP-1011) (InKline Pharmaceuticals) is being evaluated in both the treatment of idiopathic throbocytopenic purpura and inflammatory bowel disease.

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