Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood ‐onset beta‐mannosidosis

ConclusionOur findings suggest that hematopoietic cell transplant can correct the biochemical defect in β‐mannosidosis, although preservation of the neurocognitive trajectory may be a challenge.
Source: Molecular Genetics & Genomic Medicine - Category: Genetics & Stem Cells Authors: Tags: ORIGINAL ARTICLE Source Type: research

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ConclusionsMSCs transplantation proved to be a safe and tolerable therapy. Their potential therapeutic benefits were also validated. However, larger placebo controlled blinded clinical trials will be required to establish the long term safety and efficacy profile of these therapies for MS. Their translation into the clinical practice can provide a new hope for the patients of this highly debilitating disease.
Source: Multiple Sclerosis and Related Disorders - Category: Neurology Source Type: research
Multiple myeloma (MM) is an incurable plasma cell dyscrasia characterized by the uncontrollable proliferation of clonal plasma cells resulting in myeloma-defining events (end-organ damage and/or biomarkers of malignancy) [1]. MM constitutes a significant contributor to the global burden of hematological malignancy causing 2.1 million disability-adjusted life-years (DALYs) worldwide according to the Global Burden of Disease 2016 study [2]. During the last decades, an increase in the disease incidence has been clearly demonstrated, especially in North America, Western Europe and Australasia [2].
Source: Cancer Treatment Reviews - Category: Cancer & Oncology Authors: Tags: Anti-Tumour Treatment Source Type: research
Purpose of review In this review, we summarize the recently published literature that demonstrates the efficacy and safety of autologous haematopoietic stem cell therapy (AHSCT) in multiple sclerosis (MS) and highlight the importance of supportive care required for the safe and well-tolerated delivery of AHSCT. Recent findings MS is an autoimmune inflammatory and degenerative disorder of the central nervous system (CNS). In the majority of patients, the illness runs a relapsing remitting course (RRMS), culminating in a secondary progressive phase with gradual accumulation of fixed disabilities. Currently available dis...
Source: Current Opinion in Supportive and Palliative Care - Category: Palliative Care Tags: BLOOD, BONE MARROW AND LYMPHATICS: Edited by Christopher Dalley Source Type: research
ConclusionMany MS patients are considering aHSCT as a treatment option, although they think that they are not well-informed regarding aHSCT. They prefer their neurologist as a source of information. Therefore, neurologists should pro-actively inform their patients about the potential benefits and risks of aHSCT to enable them to choose the best treatment option.
Source: Multiple Sclerosis and Related Disorders - Category: Neurology Source Type: research
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Source: Journal of Neurochemistry - Category: Neuroscience Authors: Tags: Original Article Source Type: research
Chronic graft-versus-host disease (GVHD) remains a major cause of long-term morbidity and late mortality in allogeneic hematopoietic cell transplant (HCT) survivors [2,3]. Chronic GVHD is a heterogenous syndrome in which most patients have at least three involved organs (most commonly skin, mouth, liver or eyes, but also gastrointestinal tract, lung, joints, and genital tract), and treatment typically requires prolonged (median 5 years) use of immunosuppressive agents [4]. Chronic GVHD and its treatment are thus often associated with several adverse effects on long-term quality of life and functional abilities among HCT su...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
AbstractAn Italian 13-year-old boy immunosuppressed due to kidney transplant presented in November 2018 with acute flaccid paralysis with anterior horn cell involvement resembling the clinical, radiological, and laboratory features of poliomyelitis. Enterovirus was molecularly identified in cerebral spinal fluid and stool samples and the sequence analysis of the VP1 gene of enterovirus genome revealed the presence of Echovirus 30 both in CSF and in stool samples. Echovirus 30 is an emerging neurotropic virus able to cause outbreaks of aseptic meningitis and meningoencephalitis all over the world, but acute flaccid paralysi...
Source: Journal of NeuroVirology - Category: Neurology Source Type: research
Semin Neurol 2019; 39: 596-607 DOI: 10.1055/s-0039-1693008Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a relatively common autoimmune disorder affecting the peripheral nerves and nerve roots, often causing progressive or recurrent weakness with diminished reflexes. Electrodiagnostic (EDx) studies, cerebral spinal fluid (CSF) analysis, and nerve biopsy may help provide supportive evidence for the diagnosis. Most cases have a favorable response to one of the three first-line treatments: corticosteroids, IV immunoglobulin (IVIG) and plasmapheresis. Responses to these treatments may vary among individual...
Source: Seminars in Neurology - Category: Neurology Authors: Tags: Review Article Source Type: research
ConclusionsIn conclusion, PLDRH is a feasible and safe procedure with a learning curve of 65 –70 cases.
Source: Surgical Endoscopy - Category: Surgery Source Type: research
We present the reader with a brief review on the AHCT procedure, its immunomodulatory mechanism of action in MS, the most recent evidence in support of its use in patients with relapsing-remitting multiple sclerosis (RRMS), as well as its cost considerations.Recent findingsThe first meta-analysis of clinical trials of AHCT for patients with MS demonstrated durable 5-year progression-free survival rates and low treatment-related mortality. Recently, the first randomized controlled phase III clinical trial demonstrated AHCT to be superior to best available therapy for a subset of patients with RRMS. This led to the American ...
Source: Current Treatment Options in Neurology - Category: Neurology Source Type: research
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