“This is a brief report about the interim results of a study using eltrombopag (also called Promacta or, outside the United States, Revolade) to treat thrombocytopenia in patients with Wiskott Aldrich. Eltrombopag is one of the novel thrombopoietic agents; this means that unlike for example IVIG whose main effect is to interfere with platelet destruction, Eltrombo[pag stimulates making more platelets. It has been licensed in the United States since November 2008 and is now licensed in more than 50 countries worldwide. Many thousands of patients have been treated with it worldwide and it has a new oral suspension so that any age child (or adult) could take it if desired. Perhaps the biggest problem for infants and small children is that it cannot be taken within four hours of calcium so this would mean not within four hours of milk either before or after. Notwithstanding this, it has been very useful in children with ITP and there is an ongoing study that has enrolled more than 50 patients including recently 5 very young ones with more to follow. Thus far, it has generally had minimum toxicity and been generally well tolerated. One main issue with it is that patients need to be monitored for liver function testing approximately monthly initially and then every two monthly. This means that when a blood test is performed for platelets, the patients need to have drawn one more tube or one half teaspoon of blood to measure their liver tests. Very, few people have been unable to take and continue with the medication. Other side effects seem to be much rarer.

The results thus far are very encouraging in the X-linked thrombocytopenia form of Wiskott Aldrich. This is the form where the predominate issue is low platelets and it has been debated at two national meetings recently as to whether it is appropriate to do splenectomy, stem cell transplantation, gene therapy, or just supportive care. In many cases, the thrombocytopenia is the biggest problem and fixing it would resolve much of the quality of life and treatments required. Thus far on our study we have treated approximately 6 patients. The patients with the XLT form had their platelets increase substantially and had no problems with bleeding. One of them has been on the medication for more than one year and feels that it has significantly improved his life. The patients with full blown Wiskott Aldrich have generally had much less effect. This seems as if Eltrombopag might be an adjunct therapy in certain cases but is not that useful on its own. It would probably have some effect at most. However the numbers are small and additional patients treated are needed. As part of this study we are looking at platelet function in Wiskott Aldrich and there seems to be a clear effect in that regard as well. It is interesting because the WAS patients have a clear benefit in regard to platelet aggregation when stimulated via collagen (this is widespread in the body and is the underlining of blood vessels) and don’t do as well with stimulation using more “regular agents”. Therefore the primary “functional” defect appears to be that the platelets are so small and therefore that twice as many are required as normal to achieve the same degree of hemostasis (preventing bleeding).

James B. Bussel, M.D.

Professor of Pediatrics and

Professor of Pediatrics in Obstetrics and Gynecology and in Medicine

Director, Platelet Research & Treatment Program

Division of Pediatric Hematology Oncology

Weill Cornell Medical College

Contact Information: jbussel@med.cornell.edu

Contact: Dr. James B Bussel, Phone: 212-746-3474 E Mail: jbussel@med.cornell.edu

IVIG Trials - Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects.

Evaluation of the Efficacy and Safety of Flebogamma 5% DIF [Immune Globulin Intravenous (Human)] for Replacement Therapy in Pediatric Subjects With Primary Immunodeficiency Diseases.

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