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Article ; Online: Benefit of clinical pharmacists in neurology clinics at an academic medical center.

Anderson, Bethany / Snider, Melissa J / Hansen, Margaret / Parks, Caitlin / Bagnola, Aaron / Li, Junan / Freimer, Miriam / Segal, Benjamin

Journal of the American Pharmacists Association : JAPhA

2024 Volume 64, Issue 2, Page(s) 492–498.e1

Abstract: Background: Patients with neurologic diseases have complex medical needs and may benefit from the addition of clinical pharmacists in their care.: Objectives: This study aimed to describe integration and benefit of clinical pharmacists in ... ...

Abstract	Background: Patients with neurologic diseases have complex medical needs and may benefit from the addition of clinical pharmacists in their care. Objectives: This study aimed to describe integration and benefit of clinical pharmacists in neuroimmunology and neuromuscular clinics at an academic medical center. Methods: This retrospective chart review evaluated patients initiated on a neurology medication for a neuroimmunology or neuromuscular disease state before and after pharmacist integration in neurology clinics. The primary outcome measured access to an initially prescribed neuroimmunology or neuromuscular medication within 90 days of prescription. Secondary outcomes included access to an initially prescribed or alternative neurology medication owing to insurance requirements within 90 days, time from initial prescription to start, and description of pharmacist involvement. Results: There were 101 patients in the pregroup and 101 patients in the postgroup. The percentage of patients with confirmed initially prescribed medication access at 90 days increased in the postgroup compared with the pregroup (87.1% vs. 72.5%, respectively, P = 0.014). For secondary outcomes, the percentage of patients who started on an initially prescribed or alternative neuroimmunology or neuromuscular medication within 90 days also increased in the postgroup compared with the pregroup (90.0% vs. 73.3%, respectively, P = 0.004). Additional pharmacist involvement occurred in 64 patients (63.4%) in the postgroup and included prior authorization approval assistance, drug information support, and medication liaison interventions, with an average of 4.7 pharmacist interventions at each pharmacy-led encounter. Conclusion: The addition of pharmacists into neuroimmunology and neuromuscular clinics improved operational access to medications for neuroimmunology and neuromuscular conditions. In addition, pharmacists were able to assist with multiple areas of patient care including medication education, monitoring, and serving as a medication liaison. This study supports continuing to offer clinical pharmacy services in neuroimmunology and neuromuscular departments and may support the addition of clinical pharmacists into neurology services at other institutions.
MeSH term(s)	Humans ; Pharmacists ; Retrospective Studies ; Patient Care ; Pharmacy Service, Hospital ; Academic Medical Centers
Language	English
Publishing date	2024-01-19
Publishing country	United States
Document type	Journal Article
ZDB-ID	2118585-2
ISSN	1544-3450 ; 1544-3191 ; 1086-5802
ISSN (online)	1544-3450
ISSN	1544-3191 ; 1086-5802
DOI	10.1016/j.japh.2024.01.010
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Article ; Online: Recent advances in understanding and managing myasthenia gravis.

Jordan, Allison / Freimer, Miriam

F1000Research

2018 Volume 7

Abstract: Autoimmune myasthenia gravis (MG) is a neuromuscular junction disorder marked clinically by fatigable muscle weakness and serologically by the presence of autoantibodies against acetylcholine receptors (AChRs), muscle-specific kinase (MuSK), or ... ...

Abstract	Autoimmune myasthenia gravis (MG) is a neuromuscular junction disorder marked clinically by fatigable muscle weakness and serologically by the presence of autoantibodies against acetylcholine receptors (AChRs), muscle-specific kinase (MuSK), or lipoprotein-related protein 4 (LPR4). Over the past few decades, the mortality of patients with MG has seen a dramatic decline secondary to evolving interventions in critical care and medical management. In the past 2 to 3 years, there have been several changes in standard of care for the treatment of MG. These changes include confirmation of the benefit of thymectomy versus medical management alone in AChR patients and a new US Food and Drug Administration-approved medication for refractory MG. There are also several exciting new prospective drugs in the pipeline, which are in different stages of clinical trial testing.
MeSH term(s)	Animals ; Autoantibodies/immunology ; Combined Modality Therapy/methods ; Combined Modality Therapy/standards ; Disease Management ; Humans ; Myasthenia Gravis/mortality ; Myasthenia Gravis/therapy ; Salvage Therapy/methods ; Standard of Care/trends
Chemical Substances	Autoantibodies
Language	English
Publishing date	2018-10-31
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	2699932-8
ISSN	2046-1402 ; 2046-1402
ISSN (online)	2046-1402
ISSN	2046-1402
DOI	10.12688/f1000research.15973.1
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Article: Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review.

Jiang, Benjamin / Sahenk, Zarife / Satoskar, Anjali / Freimer, Miriam / Ayoub, Isabelle

Clinical nephrology

2021 Volume 96, Issue 3, Page(s) 175–179

Abstract: IgG4-related disease is an immune-mediated systemic inflammatory condition characterized by tissue infiltration of IgG4-positive plasma cells and elevated serum IgG4 concentrations. Peripheral neuropathy is an atypical manifestation of this disease. We ... ...

Abstract	IgG4-related disease is an immune-mediated systemic inflammatory condition characterized by tissue infiltration of IgG4-positive plasma cells and elevated serum IgG4 concentrations. Peripheral neuropathy is an atypical manifestation of this disease. We describe an unusual case of vasculitic neuropathy in a patient with IgG4-related kidney disease. A 55-year-old woman presented with right leg weakness progressing to bilateral leg weakness, pain and numbness of the legs, and impaired gait. She was previously evaluated for weight loss and anemia with a CT scan of the abdomen due to concern for malignancy. Abnormal enhancement of the kidneys was seen, and laboratory work-up and kidney biopsy were consistent with IgG4-related disease. Myeloperoxidase-antineutrophil cytoplasmic antibodies were also positive. In combination with the patient's asymmetric leg weakness and painful neuropathy, this raised concern for vasculitis. Sural nerve biopsy confirmed vasculitic neuropathy. Recent studies have demonstrated an overlap in the clinical characteristics of IgG4-related disease and the anti-neutrophil cytoplasmic antibody-associated vasculitides, which are known to cause vasculitic neuropathy. Clinicians should recognize this association, and IgG4-related disease should be considered in the differential diagnosis in patients with peripheral neuropathy in the right clinical context.
MeSH term(s)	Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis ; Female ; Humans ; Immunoglobulin G4-Related Disease ; Kidney ; Middle Aged ; Peripheral Nervous System Diseases/diagnosis ; Peripheral Nervous System Diseases/etiology ; Peroxidase
Chemical Substances	Peroxidase (EC 1.11.1.7)
Language	English
Publishing date	2021-08-07
Publishing country	Germany
Document type	Case Reports ; Journal Article ; Review
ZDB-ID	185101-9
ISSN	0301-0430
ISSN	0301-0430
DOI	10.5414/CN110547
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Article ; Online: Improvement of fatigue in generalised myasthenia gravis with zilucoplan.

Weiss, Michael D / Freimer, Miriam / Leite, M Isabel / Maniaol, Angelina / Utsugisawa, Kimiaki / Bloemers, Jos / Boroojerdi, Babak / Howard, Emily / Savic, Natasa / Howard, James F

Journal of neurology

2024 Volume 271, Issue 5, Page(s) 2758–2767

Abstract: Background: Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact of fatigue on MG can be assessed by Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue scale. Transformation of raw Neuro-QoL fatigue scores to T- ... ...

Abstract	Background: Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact of fatigue on MG can be assessed by Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue scale. Transformation of raw Neuro-QoL fatigue scores to T-scores is a known approach for facilitating clinical interpretation of clinically meaningful and fatigue severity thresholds. Methods: In the Phase 3, double-blind, placebo-controlled RAISE study (NCT04115293), adults with acetylcholine receptor autoantibody-positive generalised MG (MG Foundation of America Disease Class II-IV) were randomised 1:1 to daily subcutaneous zilucoplan 0.3 mg/kg or placebo for 12 weeks. Patients completing RAISE could opt to receive zilucoplan 0.3 mg/kg in an ongoing, open-label extension study, RAISE-XT (NCT04225871). In this post-hoc analysis, we evaluated the long-term effect of zilucoplan on fatigue in RAISE patients who entered RAISE-XT. We report change in Neuro-QoL Short Form Fatigue T-scores and fatigue severity levels from RAISE baseline to Week 60. Results: Mean Neuro-QoL Short Form Fatigue T-scores improved from baseline to Week 12 in the zilucoplan group (n = 86) with a clinically meaningful difference versus placebo (n = 88; least squares mean difference: - 3.61 (nominal p-value = 0.0060]), and these improvements continued further to Week 60. At Week 12, more patients on zilucoplan (n = 34, 47.2%) experienced improvements in ≥ 1 fatigue severity level from baseline versus placebo (n = 23, 28.4%; p = 0.017). At Week 60, most (n = 55, 65.5%) patients had mild fatigue or none. Conclusion: Treatment with zilucoplan demonstrated statistical and clinically meaningful improvements in fatigue scores and severity versus placebo during RAISE, which were sustained to Week 60 in RAISE-XT.
MeSH term(s)	Humans ; Myasthenia Gravis/drug therapy ; Myasthenia Gravis/complications ; Double-Blind Method ; Fatigue/etiology ; Fatigue/drug therapy ; Fatigue/physiopathology ; Male ; Female ; Adult ; Middle Aged ; Quality of Life ; Aged ; Treatment Outcome ; Severity of Illness Index ; Outcome Assessment, Health Care
Language	English
Publishing date	2024-02-24
Publishing country	Germany
Document type	Journal Article ; Randomized Controlled Trial ; Clinical Trial, Phase III
ZDB-ID	187050-6
ISSN	1432-1459 ; 0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X
ISSN (online)	1432-1459
ISSN	0340-5354 ; 0012-1037 ; 0939-1517 ; 1619-800X
DOI	10.1007/s00415-024-12209-3
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Article: Chemotherapy-induced peripheral neuropathy: an update on the current understanding.

Addington, James / Freimer, Miriam

F1000Research

2016 Volume 5

Abstract: Chemotherapy-induced peripheral neuropathy is a common side effect of selected chemotherapeutic agents. Previous work has suggested that patients often under report the symptoms of chemotherapy-induced peripheral neuropathy and physicians fail to ... ...

Abstract	Chemotherapy-induced peripheral neuropathy is a common side effect of selected chemotherapeutic agents. Previous work has suggested that patients often under report the symptoms of chemotherapy-induced peripheral neuropathy and physicians fail to recognize the presence of such symptoms in a timely fashion. The precise pathophysiology that underlies chemotherapy-induced peripheral neuropathy, in both the acute and the chronic phase, remains complex and appears to be medication specific. Recent work has begun to demonstrate and further clarify potential pathophysiological processes that predispose and, ultimately, lead to the development of chemotherapy-induced peripheral neuropathy. There is increasing evidence that the pathway to neuropathy varies with each agent. With a clearer understanding of how these agents affect the peripheral nervous system, more targeted treatments can be developed in order to optimize treatment and prevent long-term side effects.
Language	English
Publishing date	2016
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	2699932-8
ISSN	2046-1402
ISSN	2046-1402
DOI	10.12688/f1000research.8053.1
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Article ; Online: Immunoglobulin administration for the treatment of CIDP: IVIG or SCIG?

Allen, Jeffrey A / Gelinas, Deborah F / Freimer, Miriam / Runken, M Chris / Wolfe, Gil I

Journal of the neurological sciences

2019 Volume 408, Page(s) 116497

Abstract: Chronic inflammatory demyelinating polyneuropathy (CIDP) is an acquired neurological disorder characterized clinically by weakness and impaired sensory function evolving over 2 months or more, loss or significant decrease in deep tendon reflexes, and by ... ...

Abstract	Chronic inflammatory demyelinating polyneuropathy (CIDP) is an acquired neurological disorder characterized clinically by weakness and impaired sensory function evolving over 2 months or more, loss or significant decrease in deep tendon reflexes, and by electrophysiological evidence of peripheral nerve demyelination. Expeditious diagnosis and treatment of CIDP early in the disease course is critical such that irreversible disability can be avoided. Intravenous immunoglobulin (IVIG) is one first-line and maintenance therapy option for CIDP. The US Food & Drug Administration's (FDA's) approval of subcutaneous immunoglobulin (SCIG) in 2018 provides patients with CIDP more treatment options for maintenance therapy. The different options for administration of IG treatment create the need for information to assist clinicians and patients in choosing the optimal therapeutic approach. Considerations for pharmacokinetics, administration procedures, adverse events, patient variables, and cost will all be discussed in this article.
MeSH term(s)	Humans ; Immunoglobulins, Intravenous/administration & dosage ; Immunoglobulins, Intravenous/pharmacokinetics ; Infusions, Subcutaneous/methods ; Injections, Subcutaneous ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/diagnosis ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/drug therapy ; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating/metabolism ; Randomized Controlled Trials as Topic/methods
Chemical Substances	Immunoglobulins, Intravenous
Language	English
Publishing date	2019-11-09
Publishing country	Netherlands
Document type	Journal Article ; Review
ZDB-ID	80160-4
ISSN	1878-5883 ; 0022-510X ; 0374-8642
ISSN (online)	1878-5883
ISSN	0022-510X ; 0374-8642
DOI	10.1016/j.jns.2019.116497
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Article: Multidisciplinary amyloidosis care in the era of personalized medicine.

Bumma, Naresh / Kahwash, Rami / Parikh, Samir V / Isfort, Michael / Freimer, Miriam / Vallakati, Ajay / Redder, Elyse / Campbell, Courtney M / Sharma, Nidhi / Efebera, Yvonne / Stino, Amro

Frontiers in neurology

2022 Volume 13, Page(s) 935936

Abstract: Amyloidosis refers to a group of conditions where abnormal protein-or amyloid-deposits in tissues or organs, often leading to organ malfunction. Amyloidosis affects nearly any organ system, but especially the heart, kidneys, liver, peripheral nervous ... ...

Abstract	Amyloidosis refers to a group of conditions where abnormal protein-or amyloid-deposits in tissues or organs, often leading to organ malfunction. Amyloidosis affects nearly any organ system, but especially the heart, kidneys, liver, peripheral nervous system, and gastrointestinal tract. Neuromuscular deficits comprise some of its ubiquitous manifestations. Amyloidosis can be quite challenging to diagnose given its clinical heterogeneity and multi-system nature. Early diagnosis with accurate genetic and serologic subtyping is key for effective management and prevention of organ decline. In this review, we highlight the value of a multidisciplinary comprehensive amyloidosis clinic. While such a model exists at numerous clinical and research centers across the globe, the lack of more widespread adoption of such a model remains a major hindrance to the timely diagnosis of amyloidosis. Such a multidisciplinary care model allows for the timely and effective diagnosis of amyloidosis, be it acquired amyloid light amyloidosis (AL), hereditary transthyretin amyloidosis (hATTR), or wild type amyloidosis (TTR-wt), especially in the current era of personalized genomic medicine. A multidisciplinary clinic optimizes the delivery of singular or combinatorial drug therapies, depending on amyloid type, fibril deposition location, and disease progression. Such an arrangement also helps advance research in the field. We present our experience at The Ohio State University, as one example out of many, to highlight the centrality of a multi-disciplinary clinic in amyloidosis care.
Language	English
Publishing date	2022-10-13
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2564214-5
ISSN	1664-2295
ISSN	1664-2295
DOI	10.3389/fneur.2022.935936
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Article: Beta-Adrenergic Antagonist Tolerance in Amyloid Cardiomyopathy.

Ramsell, Stuart / Arias Bermudez, Carlos / Takem Baiyee, Cyril Ayuk Mbeng / Rodgers, Brandon / Parikh, Samir / Almaani, Salem / Sharma, Nidhi / LoRusso, Samantha / Freimer, Miriam / Redder, Elyse / Bumma, Naresh / Vallkati, Ajay / Efebera, Yvonne / Kahwash, Rami / Campbell, Courtney M

Frontiers in cardiovascular medicine

2022 Volume 9, Page(s) 907597

Abstract: Background: Beta-adrenergic antagonists or blockers (BB) are a cornerstone of cardiac therapy for multiple indications. However, BB are considered relatively contraindicated in amyloid cardiomyopathy due to poor tolerance. This intolerance is ... ...

Abstract	Background: Beta-adrenergic antagonists or blockers (BB) are a cornerstone of cardiac therapy for multiple indications. However, BB are considered relatively contraindicated in amyloid cardiomyopathy due to poor tolerance. This intolerance is hypothesized to be due to concomitant neuropathy and significant restrictive cardiomyopathy. This study analyzes the incidence and characteristics of BB tolerance in patients with amyloid cardiomyopathy. Methods: Through a single-center retrospective chart review, patients with amyloid cardiomyopathy, confirmed by endomyocardial biopsy or technetium-99 pyrophosphate scan, were identified and clinical data was collected. Statistical methods included Chi-square test and two sample Results: Of 135 cardiac amyloidosis patients, 27 patients (20.0%) had no BB use, 56 patients (41.5%) were current BB users, and 52 patients (38.5%) were prior BB users. The most frequent indications for BB use were heart failure, hypertension, coronary artery disease, and arrhythmia. The most common reason for stopping BB therapy was hypotension (62.8%) followed by fatigue, bradycardia, and orthostasis. Neurologic symptoms at the initial BB prescription or most recent evaluation were not significantly different between current and prior BB users. Their cardiovascular profiles were similar by ejection fraction, wall thickness, troponin I, and brain natriuretic peptide. There was no association for BB discontinuation based on amyloid subtype, sex, or race. Conclusion: The majority of patients with amyloid cardiomyopathy were prescribed BB, and over half of these patients still tolerated BB therapy. Current and prior BB users had similar profiles from a cardiovascular and neurologic perspective, with no association identified to predict BB discontinuation.
Language	English
Publishing date	2022-07-11
Publishing country	Switzerland
Document type	Journal Article
ZDB-ID	2781496-8
ISSN	2297-055X
ISSN	2297-055X
DOI	10.3389/fcvm.2022.907597
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Article: Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study.

Howard, James F / Bresch, Saskia / Farmakidis, Constantine / Freimer, Miriam / Genge, Angela / Hewamadduma, Channa / Hinton, John / Hussain, Yessar / Juntas-Morales, Raul / Kaminski, Henry J / Maniaol, Angelina / Mantegazza, Renato / Masuda, Masayuki / Nowak, Richard J / Sivakumar, Kumaraswamy / Śmiłowski, Marek / Utsugisawa, Kimiaki / Vu, Tuan / Weiss, Michael D /

Zajda, Małgorzata / Bloemers, Jos / Boroojerdi, Babak / Brock, Melissa / de la Borderie, Guillemette / Duda, Petra W / Vanderkelen, Mark / Leite, M Isabel

Therapeutic advances in neurological disorders

2024 Volume 17, Page(s) 17562864241243186

Abstract: Background: Generalized myasthenia gravis (gMG) is a chronic, unpredictable disease associated with high treatment and disease burdens, with a need for more effective and well-tolerated treatments.: Objectives: To evaluate the long-term safety, ... ...

Abstract	Background: Generalized myasthenia gravis (gMG) is a chronic, unpredictable disease associated with high treatment and disease burdens, with a need for more effective and well-tolerated treatments. Objectives: To evaluate the long-term safety, tolerability, and efficacy of zilucoplan in a mild-to-severe, acetylcholine receptor autoantibody-positive (AChR+) gMG population. Design: Ongoing, multicenter, phase III open-label extension (OLE) study. Methods: Eligible patients had completed a qualifying randomized, placebo-controlled phase II or phase III zilucoplan study and received daily, self-administered subcutaneous 0.3 mg/kg zilucoplan. The primary endpoint was incidence of treatment-emergent adverse events (TEAEs). Secondary efficacy endpoints included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Results: In total, 200 patients enrolled. At the cut-off date (8 September 2022), median (range) exposure to zilucoplan in RAISE-XT was 1.2 (0.11-4.45) years. Mean age at OLE baseline was 53.3 years. A total of 188 (94%) patients experienced a TEAE, with the most common being MG worsening ( Conclusion: Zilucoplan demonstrated a favorable long-term safety profile, good tolerability, and sustained efficacy through to Week 60 with consistent benefits in a broad AChR+ gMG population. Additional long-term data will be available in future analyses. Trial registration: ClinicalTrials.gov identifier: NCT04225871 (https://clinicaltrials.gov/ct2/show/NCT04225871).
Language	English
Publishing date	2024-04-17
Publishing country	England
Document type	Journal Article
ZDB-ID	2442245-9
ISSN	1756-2864 ; 1756-2856
ISSN (online)	1756-2864
ISSN	1756-2856
DOI	10.1177/17562864241243186
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Article ; Online: Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy.

Annals of clinical and translational neurology

2023 Volume 10, Issue 5, Page(s) 686–695

Abstract: Valosin-containing protein (VCP)-associated multisystem proteinopathy (MSP) is a rare genetic disorder with abnormalities in the autophagy pathway leading to various combinations of myopathy, bone diseases, and neurodegeneration. Ninety percent of ... ...

Abstract	Valosin-containing protein (VCP)-associated multisystem proteinopathy (MSP) is a rare genetic disorder with abnormalities in the autophagy pathway leading to various combinations of myopathy, bone diseases, and neurodegeneration. Ninety percent of patients with VCP-associated MSP have myopathy, but there is no consensus-based guideline. The goal of this working group was to develop a best practice set of provisional recommendations for VCP myopathy which can be easily implemented across the globe. As an initiative by Cure VCP Disease Inc., a patient advocacy organization, an online survey was initially conducted to identify the practice gaps in VCP myopathy. All prior published literature on VCP myopathy was reviewed to better understand the different aspects of management of VCP myopathy, and several working group sessions were conducted involving international experts to develop this provisional recommendation. VCP myopathy has a heterogeneous clinical phenotype and should be considered in patients with limb-girdle muscular dystrophy phenotype, or any myopathy with an autosomal dominant pattern of inheritance. Genetic testing is the only definitive way to diagnose VCP myopathy, and single-variant testing in the case of a known familial VCP variant, or multi-gene panel sequencing in undifferentiated cases can be considered. Muscle biopsy is important in cases of diagnostic uncertainty or lack of a definitive pathogenic genetic variant since rimmed vacuoles (present in ~40% cases) are considered a hallmark of VCP myopathy. Electrodiagnostic studies and magnetic resonance imaging can also help rule out disease mimics. Standardized management of VCP myopathy will optimize patient care and help future research initiatives.
MeSH term(s)	Humans ; Valosin Containing Protein/genetics ; Muscular Diseases/diagnosis ; Muscular Diseases/genetics ; Muscular Diseases/therapy ; Muscular Dystrophies, Limb-Girdle/diagnosis ; Muscular Dystrophies, Limb-Girdle/genetics ; Muscular Dystrophies, Limb-Girdle/therapy ; Phenotype ; Proteostasis Deficiencies
Chemical Substances	Valosin Containing Protein (EC 3.6.4.6) ; VCP protein, human (EC 3.6.4.6)
Language	English
Publishing date	2023-04-07
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	2740696-9
ISSN	2328-9503 ; 2328-9503
ISSN (online)	2328-9503
ISSN	2328-9503
DOI	10.1002/acn3.51760
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