Sporadic late onset nemaline myopathy with monoclonal gammopathy of undetermined significance: two cases with long term stability
| Autor: | Kaveh Samii, Johannes Alexander Lobrinus, André Truffert, Ruxandra Iancu Ferfoglia, André Kohler |
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| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
Pediatrics medicine.medical_specialty sporadic late-onset nemaline myopathy medicine.medical_treatment lenalidomide Cardiomyopathy lcsh:Medicine Late onset Muscle disorder ddc:616.07 stem cell transplantation Article lcsh:QM1-695 03 medical and health sciences 0302 clinical medicine Nemaline myopathy medicine Orthopedics and Sports Medicine Myopathy Molecular Biology Lenalidomide ddc:616 Chemotherapy business.industry lcsh:R lcsh:Human anatomy Cell Biology medicine.disease ddc:616.8 030104 developmental biology Neurology (clinical) medicine.symptom business 030217 neurology & neurosurgery Monoclonal gammopathy of undetermined significance medicine.drug monoclonal gammopathy of undetermined significance |
| Zdroj: | European Journal of Translational Myology European Journal of Translational Myology, Vol. 30, No 3 (2020) P. 9225 European Journal of Translational Myology (2020) |
| ISSN: | 2037-7452 |
| Popis: | Monoclonal gammopathy of undetermined significance (MGUS) associated to sporadic late onset nemaline myopathy (SLONM) is a rare and severely disabling condition of quickly progressive limb girdle acquired myopathy. It is believed by some authors to be due to myotoxicity of light chain deposits. Two female patients were diagnosed with MGUS associated SLONM. In the first case, diagnosis was delayed by 6 years thus giving time for a severe generalized myopathy and cardiomyopathy to develop. A single anti-myeloma chemotherapy with lenalidomide markedly improved and stabilized the patient’s condition despite respiratory and cardiac insufficiency. In our second patient the condition was identified one year after onset of the first symptom and markedly improved after autologous bone marrow transplantation and lenalidomide. Clinicians should be aware of monoclonal gammopathy associated sporadic late onset nemaline myopathy as this acquired muscle disorder, although extremely rare, may be reversed by adequate management. |
| Databáze: | OpenAIRE |
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