Pomalidomide – a cancer medicine was found in the clinical trial as being relatively safe and serving its usefulness in the management of hereditary hemorrhagic telangiectasia (HHT), a bleeding disorder occurring in excess of 1/5,000 populations globally.
This trial was performed by Keith McCrae, M. D from Cleveland Clinic and it was stopped early because of such positive results and was published in New England Journal of Medicine. The idea for this trial was established on the basis of one patient.
HHT is a disease whereby superior-level vessels are coiled and spiraled in an improper manner. This results in massive nasal bleeding that is characteristic of the disease to be under control. It also causes gastrointestinal hemorrhage and fatal outcomes involving lungs, liver and brain.
Thus, Dr. McCrae began seeking for another better treatment and it was realized that thalidomide, which is a cancer drug, has some impact on some of the patients with the same diseases.
He administered a small portion of the said drug to his patient and within two to three weeks the bleeding had decreased by seventy five percent. The doctor prescribed thalidomide on the other patients who also showed similar HHT symptoms, and they also improved. ‘It was amazing. ‘ Dr. McCrae smiled and looked a little more pleased with himself.
One was very infrequent and I saw something like that in my own clinical practice and I thought we should learn it.Thalidomide targets multiple myeloma, is very risky; instead of searching again using thalidomide, Dr. McCrae used pomalidomide, an FDA approved drug for bone marrow cancer.
He tried pomalidomide for the first time in a pilot study and the drug seemed to be effective and had no toxicity. To assess the efficacy of pomalidomide, 144 patients with HHT were enrolled randomly across 11 U. S. centres between November 2019 and June 2023.The patient advocacy organization known as CureHHT shall make sure that the patients are advised to join the clinical trial.
All the participants had mild to severe septra-nasal bleed and required either IM iron preparations or blood products. The data coordinating centre for the conduct of the study was RTI International based in North Carolina and the clinical coordinating centre was C5 Research based in Cleveland Clinic ARO.
Patients with HHT treated with pomalidomide experience less severe and frequent nosebleeds, reduced need for blood as well as iron transfusion and improved quality of life. A first early planned analysis of the data that took place at June 2023 points towards the effectiveness of pomalidomide and thus the trial were stopped.
Possible assertion by pomalidomide is the inhibition of angiogenesis in proliferation of anomalous blood vessels. Nevertheless, the treatment of this disorder has been successfully identified, but there are many aspects of the functioning of HHT. For additional information on this disease and pomalidomide as well as other drugs’ effects on HHT, Dr. McCrae is hoping to, they would hopefully get more funding.
Reference:
Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia, New England Journal of Medicine (2024).
