Have A Goal To Taper Off Your Use Of Steroids? Many Clinical Studies For New Drugs Share The Same Mission
Ongoing clinical trials are paving the way for reducing steroid dependency and improving treatment options for various medical conditions.
For many people living with rare diseases, the reality is that there is no approved therapy to treat their condition. However, many autoimmune and inflammatory conditions offer one treatment option: corticosteroids (more commonly known as “steroids.”). Corticosteroids are mainly used to reduce inflammation and suppress the immune system, which makes them a valuable treatment option for patients who are in need of these effects.
People with neuromuscular conditions like myasthenia gravis and autoimmune myositis may take high doses of corticosteroids to allow them to manage activities in their daily lives. Oral corticosteroid tablets are the most powerful type of steroids—they can affect the entire body, and the higher the dose and the length of time that they’re taken, the greater the risk of side effects. Oral steroids taken over a period of time (longer than three weeks) may lead to even more significant side effects including weight gain, acne, mood changes, thin skin, osteoporosis, and mental health complications.
As a result, one of the goals shared by both patients and doctors is to reduce the daily intake of steroids in order to avoid these side effects, without causing an escalation of the disease. In many clinical studies for new medications, reduction or elimination of steroids is named as one of the goals.
During the course of the study, under the careful observation of the clinicians, the steroid dosage can be reduced if the patient is showing improvement in disease signs or symptoms. (The steroids will only be reduced if the doctor’s assessment confirms it will be safe to do so, as is the case with the specialist who normally treats the patient.) Doctors base their process for lowering steroid doses on evidence from other studies, or guidelines designed by experts who evaluate multiple studies to determine the best course.
Neurologists determine the success of a study drug in lowering steroid use in neurological cancers by following the recommendations of Neuro-Oncology (RANO) Working Group, which recommends the following criteria as a “response” to the study medication in reducing steroid use:
“Responders” in a clinical study means they would be able to reduce their total daily corticosteroid dose by half, for at least one week, and still have their disease status be considered “stable”, or improved. This must be sustained for at least 4 weeks after the initial assessment, in order to be considered a ‘response’. (Source: 1.Arvod, ND et al. Neuro Oncol. 2018 Jun; 20(7): 897–906)
Among 28 studies for new systemic lupus therapies, all of the therapies in the clinical trials were added to the participant’s existing steroid therapy. An analysis of these 28 studies showed that most therapies in the trials were assessed to see if steroid doses could be reduced. In most of the trials, the steroid dose could be lowered.
(Source: Oon S, et al. Meta-Analysis Semin Arthritis Rheum. 2018 Oct;48(2):221-239. doi: 10.1016/j.semarthrit.2018.01.001. Epub 2018 Jan 6.)
In another study involving 35 participants with myasthenia gravis (MG), methotrexate was added to reduce their doses of daily oral prednisone. (Source: Li D, et al. Front. Immunol., 18 March 2022, Sec. Multiple Sclerosis and Neuroimmunology Volume 13 - 2022 | https://doi.org/10.3389/fimmu.2022.839075 )
Currently there are clinical studies for myositis and MOGAD, where the investigational drug is added to the participants’ regular steroid dose, and one of the goals of these studies is to determine whether the addition of the study drug can allow for the reduction of the dose of daily oral steroids.
For people managing rare disease symptoms, corticosteroids often emerge as a lifeline—but one with potentially debilitating side effects. Many clinical trials now aim to reduce steroid reliance, offering new hope for streamlined treatment with reduced side effects.
If you or someone you care for has myositis or MOGAD and is interested in clinical studies with a goal of reducing steroid dosage, contact us at support@knowrare.com.
