Proximal muscular atrophy and weakness: An unusual adverse effect of deferasirox iron chelation therapy
| Autor: | Katharina Vill, V. Teusch, Wolfgang Müller-Felber, M.H. Albert, Astrid Blaschek, Lucia Gerstl, S. Huetker |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
medicine.medical_specialty
Pediatrics Weakness Iron Iron Chelating Agents Benzoates 03 medical and health sciences 0302 clinical medicine Atrophy medicine Humans Transplantation Homologous Child Adverse effect Genetics (clinical) Muscle Weakness business.industry Standard treatment beta-Thalassemia Deferasirox Hematopoietic Stem Cell Transplantation Beta thalassemia Twins Monozygotic Triazoles medicine.disease Chelation Therapy Surgery Discontinuation Transplantation Muscular Atrophy Neurology Child Preschool 030220 oncology & carcinogenesis Pediatrics Perinatology and Child Health Female Neurology (clinical) medicine.symptom business 030215 immunology medicine.drug |
| Zdroj: | Neuromuscular Disorders. 26:322-325 |
| ISSN: | 0960-8966 |
| DOI: | 10.1016/j.nmd.2016.02.011 |
| Popis: | Deferasirox is a standard treatment for chronic transfusional iron overload. Adverse effects of deferasirox have been reported in large prospective studies. We report two cases of monozygotic twins manifesting with proximal muscular atrophy and weakness under deferasirox. Discontinuation of deferasirox resulted in symptom improvement and ultimately in complete remission five months after successful haematopoietic stem cell transplantation. Broad diagnostic work-up could not bring evidence of another aetiology of muscular weakness. Iron overload or beta thalassemia itself as a cause is considered unlikely in our patients because the chronological coincidence of muscular symptoms was contra-directional to serum ferritin levels and significant clinical improvement was observed promptly after cessation of deferasirox even before transplantation. These observations suggest that the development of muscular weakness in patients on deferasirox should be recognised as a possible adverse effect of the drug. |
| Databáze: | OpenAIRE |
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