Journal of Shandong University (Health Sciences) ›› 2022, Vol. 60 ›› Issue (10): 74-81.doi: 10.6040/j.issn.1671-7554.0.2022.0084

Previous Articles     Next Articles

Three cases of sporadic late-onset nemaline myopathy with monoclonal gammopathy and literature review

DONG Wenhao1, ZHAO Bing2, LI Zhao1, ZHANG Chen1, YUAN Chenglu1   

  1. 1. Department of Hematology;
    2. Department of Neurology, Qilu Hospital(Qingdao), Cheeloo College of Medicine, Shandong University, Qingdao 266035, Shandong, China
  • Published:2022-09-30

PDF (PC)

201

Abstract: Objective Three patients suspected of sporadic late-onset nemaline myopathy with monoclonal gammopathy were diagnosed by muscle pathological examination and monoclonal protein screening, so as to improve the understanding of this rare disease and explore its diagnosis and treatment. Methods The characteristics of this disease were summarized and relevant literatures were reviewed. Results All of the 3 patients presented with progressively aggravated muscle weakness, which was diagnosed by muscle biopsy as sporadic late-onset nemaline myopathy with monoclonal gammopathy. Autologous hematopoietic stem cell transplantation was effective. Conclusion Sporadic late-onset nemaline myopathy is a rare, subacute progressive myopathy characterized by proximal muscle weakness and atrophy, often complicated with monoclonal gammopathy. Therapies directed at eradication of M protein are effective.

Key words: Monoclonal gammopathy, Sporadic late-onset nemaline myopathy, Autologous hematopoietic stem cell transplantation

CLC Number: 

  • R746.9
[1] Tanboon J, Uruha A, Arahata Y, et al. Inflammatory features in sporadic late-onset nemaline myopathy are independent from monoclonal gammopathy[J]. Brain Pathol, 2021, 31(3): e12962. doi: 10.1111/bpa.12962.
[2] Uruha A, Benveniste O. Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance[J]. Curr Opin Neurol, 2017, 30(5): 457-463.
[3] Chahin N, Selcen D, Engel AG. Sporadic late onset nemaline myopathy[J]. Neurology, 2005, 65(8): 1158-1164.
[4] Belhomme N, Maamar A, Le Gallou T, et al. Rare myopathy associated to MGUS, causing heart failure and responding to chemotherapy[J]. Ann Hematol, 2017, 96(4): 695-696.
[5] Schnitzler LJ, Schreckenbach T, Nadaj-Pakleza A, et al. Sporadic late-onset nemaline myopathy: clinico-pathological characteristics and review of 76 cases[J]. Orphanet J Rare Dis, 2017, 12(1): 86.
[6] Berentsen S, Ulvestad E, Langholm R, et al. Primary chronic cold agglutinin disease: a population based clinical study of 86 patients[J]. Haematologica, 2006, 91(4): 460-466.
[7] Daoud MS, Lust JA, Kyle RA, et al. Monoclonal gammopathies and associated skin disorders[J]. J Am Acad Dermatol, 1999, 40(4): 507-535.
[8] Leung N, Bridoux F, Hutchison CA, et al. Monoclonal gammopathy of renal significance: when MGUS is no longer undetermined or insignificant[J]. Blood, 2012, 120(22): 4292-4295.
[9] Fermand JP, Bridoux F, Kyle RA, et al. How I treat monoclonal gammopathy of renal significance(MGRS)[J]. Blood, 2013, 122(22): 3583-3590.
[10] Eskazan A E, Gunduz A. What is the best treatment approach for sporadic late-onset nemaline myopathy associated with “monoclonal gammopathy of neurological significance”?[J]. Int J Cancer, 2021, 148(11): 2638-2639.
[11] Van De Donk NW, Palumbo A, Johnsen HE, et al. The clinical relevance and management of monoclonal gammopathy of undetermined significance and related disorders: recommendations from the European Myeloma Network[J]. Haematologica, 2014, 99(6): 984-996.
[12] Naddaf E, Milone M, Kansagra A, et al. Sporadic late-onset nemaline myopathy: Clinical spectrum, survival, and treatment outcomes [J]. Neurology, 2019, 93(3): e298-e305.
[13] Voermans NC, Minnema M, Lammens M, et al. Sporadic late-onset nemaline myopathy effectively treated by melphalan and stem cell transplant[J]. Neurology, 2008, 71(7): 532-534.
[14] Voermans NC, Benveniste O, Minnema MC, et al. Sporadic late-onset nemaline myopathy with MGUS: long-term follow-up after melphalan and SCT[J]. Neurology, 2014, 83(23): 2133-2139.
[15] 张包静子, 岳冬曰, 高名士,等 成人晚发型杆状体肌病合并单克隆丙种球蛋白病1例报道及文献复习[J]. 中国临床神经科学, 2015, 23(5): 509-515.
[16] Kotchetkov R, Susman D, Bhutani D, et al. Chemotherapy-based approach is the preferred treatment for sporadic late-onset nemaline myopathy with a monoclonal protein[J]. Int J Cancer, 2021, 148(11): 2807-2814.
[17] Benveniste O, Laforet P, Dubourg O, et al. Stem cell transplantation in a patient with late-onset nemaline myopathy and gammopathy[J]. Neurology, 2008, 71(7): 531-532.
[18] Milone M, Katz A, Amato AA, et al. Sporadic late onset nemaline myopathy responsive to IVIg and immunotherapy[J]. Muscle Nerve, 2010, 41(2): 272-276.
[19] Novy J, Rosselet A, Spertini O, et al. Chemotherapy is successful in sporadic late onset nemaline myopathy(SLONM)with monoclonal gammopathy[J]. Muscle Nerve, 2010, 41(2): 286-287.
[20] Doppler K, Knop S, Einsele H, et al. Sporadic late onset nemaline myopathy and immunoglobulin deposition disease[J]. Muscle Nerve, 2013, 48(6): 983-988.
[21] Montagnese F, Portaro S, Musumeci O, et al. Sporadic late-onset nemaline myopathy in a woman with multiple myeloma successfully treated with lenalidomide/dexamethasone[J]. Muscle Nerve, 2015, 51(6): 934-935.
[22] Maeda MH, Ohta H, Izutsu K, et al. Sporadic late-onset nemaline myopathy as a rare cause of slowly progressive muscle weakness with young adult onset[J]. Muscle Nerve, 2015, 51(5): 772-774.
[23] Belkhribchia MR, Tazi I, Louhab N, et al. Autologous stem cell transplantation in a patient with sporadic late-onset nemaline myopathy and monoclonal gammopathy: First Moroccan experience[J]. Presse Med, 2017, 46(1): 122-125.
[24] Kotchetkov R, Dyszkiewicz-Korpanty A, Kukreti V. Chemotherapy with stem cell transplantation is more effective than immunotherapy in sporadic late onset nemaline myopathy with monoclonal gammopathy[J]. Bone Marrow Transplant, 2018, 53(7): 895-899.
[25] Monforte M, Primiano G, Silvestri G, et al. Sporadic late-onset nemaline myopathy: clinical, pathology and imaging findings in a single center cohort[J]. J Neurol, 2018, 265(3): 542-551.
[26] Okhovat AA, Nilipour Y, Boostani R, et al. Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance: Report of four patients[J]. Neuromuscul Disord, 2021, 31(1): 29-34.
[27] Broch K, Popperud T, Gude E, et al. A middle-aged man presenting with progressive heart failure, myopathy, and monoclonal gammopathy of uncertain significance[J]. JACC Case Rep, 2020, 2(5): 785-789.
[28] Truffert A, Iancu Ferfoglia R, Lobrinus JA, et al. Sporadic late onset nemaline myopathy with monoclonal gammopathy of undetermined significance: two cases with long term stability[J]. Eur J Transl Myol, 2020, 30(3): 9225.
[29] Maia T, Bergantim R, Costa H, et al. Impact of hematologic complete response in the treatment of sporadic late-onset nemaline myopathy associated with monoclonal gammopathy[J]. Clin Case Rep, 2021, 9(7): e04471. doi: 10.1002/ccr3.4471.
[30] Ando T, Sato T, Kurahashi S, et al. A case of sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance: long-term observation of neurological symptoms after autologous stem-cell transplantation[J]. Nagoya J Med Sci, 2021, 83(3): 641-647.
[1] GENG Hongzhi, LI Wei, YAN Chuanzhu, LYU Xiaoqing, LIN Pengfei. Clinical, muscle pathological and genetic analyses of a centronuclear myopathy pedigree with a mutation in MTM1 gene [J]. Journal of Shandong University (Health Sciences), 2020, 58(6): 1-7.
[2] REN Hong, WANG Wei, LIN Yan, LOU Jianwei, JI Kunqian, ZHAO Yuying, YAN Chuanzhu. A case report of Barth syndrome [J]. Journal of Shandong University (Health Sciences), 2022, 60(4): 123-127.
Viewed
Full text


Abstract

Cited
  Shared   
  Discussed   
[1] SUO Dongyang, SHEN Fei, GUO Hao, LIU Lichang, YANG Huimin, YANG Xiangdong. Expression and mechanism of Tim-3 in animal model of drug-induced acute kidney injury[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 1 -6 .
[2] MA Qingyuan, PU Peidong, HAN Fei, WANG Chao, ZHU Zhoujun, WANG Weishan, SHI Chenhui. Effect of miR-27b-3p regulating SMAD1 on osteosarcoma cell proliferation, migration and invasion[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 32 -37 .
[3] ZHANG Baowen, LEI Xiangli, LI Jinna, LUO Xiangjun, ZOU Rong. miR-21-5p targeted TIMP3 to inhibit proliferation and extracellular matrix accumulation of mesangial cells in Type II diabetic nephropathy mice[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 7 -14 .
[4] FU Jieqi, ZHANG Man, ZHANG Xiaolu, LI Hui, CHEN Hong. Molecular mechanism of Toll-like receptor 4 in the aggravation of blood lipid accumulation by inhibiting the peroxisome proliferator-activate receptor γ[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 24 -31 .
[5] LONG Tingting, XIE Ming, ZHOU Lu, ZHU Junde. Effect of Noggin protein on learning and memory abilities and the dentate gyrus structure after cerebral ischemia reperfusion injury in mice[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 15 -23 .
[6] LI Ning, LI Juan, XIE Yan, LI Peilong, WANG Yunshan, DU Lutao, WANG Chuanxin. Expression of LncRNA AL109955.1 in 80 cases of colorectal cancer and its effect on cell proliferation, migration and invasion[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 38 -46 .
[7] XU Yuxiang, LIU Yudong, ZHANG Peng, DUAN Ruisheng. A retrospective analysis of risk factors of cerebral microbleeds in 101 patients with cerebral small vessel disease[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 67 -71 .
[8] DING Xiangyun, YU Qingmei, ZHANG Wenfang, ZHUANG Yuan, HAO Jing. Correlation of the expression of insulin-like growth factor II in granulosa cells and ovulation induction outcomes of 84 patients with polycystic ovary syndrome[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 60 -66 .
[9] SHI Shuang, LI Juan, MI Qi, WANG Yunshan, DU Lutao, WANG Chuanxin. Construction and application of a miRNAs prognostic risk assessment model of gastric cancer[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 47 -52 .
[10] LYU Longfei, LI Lin, LI Shuhai, QI Lei, LU Ming, CHENG Chuanle, TIAN Hui. Application of laparoscopic fine needle catheter jejunostomy in minimally invasive McKeown resection of esophageal cancer[J]. Journal of Shandong University (Health Sciences), 2020, 1(7): 77 -81 .
Copyright 2018 © Editorial office of Journal of Shandong University (Health Sciences)
Supported by Beijing Magtech Co.Ltd