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Case Reports
. 2021 Jul 16;9(7):e04471.
doi: 10.1002/ccr3.4471. eCollection 2021 Jul.

Impact of hematologic complete response in the treatment of sporadic late-onset nemaline myopathy associated with monoclonal gammopathy

Tânia Maia  1 , Rui Bergantim  1   2   3   4 , Henrique Costa  5 , Jorge Pinheiro  6 , Fernanda Trigo  1
Affiliations
Case Reports

Impact of hematologic complete response in the treatment of sporadic late-onset nemaline myopathy associated with monoclonal gammopathy

Tânia Maia et al. Clin Case Rep. .

Abstract

Monoclonal gammopathy of undetermined significance (MGUS) may be associated with pathologies with severe neuromuscular manifestations such as sporadic late-onset nemaline myopathy (SLONM). We describe a difficult to diagnose case of SLNOM with marked clinical improvement after achieving gammopathy complete hematologic response.

Keywords: autologous stem cell transplant; monoclonal gammopathy of undetermined significance; sporadic late‐onset nemaline myopathy.

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Conflict of interest statement

RB: grants from Celgene, SPH/APCL/Amgen; advisory board and speaker honoraria from Celgene, Janssen, Takeda and Amgen. FT: grant SPH/APCL/Amgen; advisory board and speaker honoraria from Celgene, Janssen, Takeda and Amgen.

Figures

FIGURE 1
FIGURE 1
(A) Dropped head and proximal dominant limb weakness with kyphotic posture sustained before autologous stem cell transplant. (B) Notable amelioration of cervical muscular force, not adopting a kyphotic posture, after cyclophosphamide, bortezomib, and dexamethasone
FIGURE 2
FIGURE 2
Muscle biopsy with marked variation in fiber size and clusters of intracytoplasmic rods, often filling atrophic fibers, with no inflammatory cells, are the pathological hallmark. (A) Gomori 100×; (B) Gomori 400×; (C) Myotilin Immunohistochemistry 200×; (D) Electron dense rod in electron microscopy
FIGURE 3
FIGURE 3
Bodyweight and muscle strength variation during clinical evolution and treatments. (ASCT, autologous transplant; CyBorDex, cyclophosphamide, bortezomib, dexamethasone; DTR, deep tendon reflexes; Flex/ext, cervical flexion/extension; Hyper, hyperreflexia; IMNF, immunofixation in peripheral blood; Kg, kilograms; Len/dex, lenalidomide and dexamethasone; LL, lower limb; N, normal; Prox, proximal; Resp ins, respiratory insufficiency; Rot R/L, cervical rotation to the right/to the left; UL, upper limb); Strength measured according to MRC (Medical Research Council) scale. Blue line: body weigh variation; orange shaded area: immunofixation negativity; green line: treatments.
See this image and copyright information in PMC

References

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