Nonmyeloablative Autologous Hematopoietic Stem Cell Transplantation for Refractory Cidp

Oyama, Yu; Sufit, Robert; Loh, Yvonne; Statkute, Laisvyde; Yaung, Kimberly; Quigley, Kathleen; Gonda, E.; Spahovic, Dzemila; Bronesky, D.; Burt, Richard K.

doi:10.1212/01.wnl.0000277266.53449.8b

Cited by 46 publications

(35 citation statements)

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“…Around 70-80% of patients with CIDP respond to first line immunomodulatory treatments including corticosteroids, IVIg, and plasma exchange. A number of retrospective reports from Europe and North America showed that the use of autologous HSCT in patients who failed standard treatment resulted in drug free remissions [92][93][94]. A phase II trial Haematopoietic stem cell transplantation in chronic inflammatory demyelinating polyneuropathy is currently recruiting [95].…”

Section: Other Neurological Diseasesmentioning

confidence: 99%

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Kelsey

Oliveira

Badoglio

et al. 2016

Current Research in Translational Medicine

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Section: Other Neurological Diseasesmentioning

confidence: 99%

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Kelsey

Oliveira

Badoglio

et al. 2016

Current Research in Translational Medicine

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“…Alemtuzumab is expensive, with our hospital billing about $1,700 for each infusion. & Stem cell transplantation has been reported to be effective in treatment-resistant CIDP [47][48][49]. However, because of the severe immunosuppression, it should be considered only as a last treatment option for patients with severe CIDP who have been unresponsive to multiple other therapies or who have suffered intolerable side effects from more conventional treatments.…”

Section: Emerging Therapiesmentioning

confidence: 99%

Inflammatory Demyelinating Neuropathies

Lopate

Pestronk

2011

Curr Treat Options Neurol

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The primary goal of therapy in patients with the Guillain-Barré syndrome (GBS) and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is improved strength and functional ability. Improvement in pain, sensory loss, gait disorder, and autonomic instability are other goals of therapy. Patients with very mild symptoms that do not interfere with activities of daily living can be observed for deterioration without treatment. For GBS, standard care includes plasma exchange (PE) or human immune globulin (HIG), both of which have similar efficacy. Supportive care in the intensive care unit may be needed for those patients with severe bulbar or respiratory weakness. We treat most patients with PE, usually performing an exchange every other day for a total of five exchanges. We use HIG in children, if there are antiglycolipid antibodies (eg, anti-GM1 or anti-GQ1b) or if there is a contraindication to PE, such as hemodynamic instability; severe renal, hepatic, or cardiac disease; or poor venous access. For CIDP, there are no guidelines concerning the initial choice of therapy. Corticosteroids, HIG, and PE have all been shown to be effective in prospective, randomized controlled trials, and comparison trials have shown equal efficacy among these three immunomodulating therapies. The choice of therapy depends on several factors including disease severity, concomitant illnesses, side-effect profile, potential drug interactions, venous access, age-related risks, and cost of treatment. In patients with moderate to severe symptoms, treatment with corticosteroids or HIG should be used. We usually use high-dose, intermittent methylprednisolone as the initial drug of choice. We believe intermittent corticosteroids are better than HIG because of their good safety profile, low cost, ease of administration (can be given intravenously or by mouth), and proven efficacy. If there is a major contraindication to corticosteroids, then HIG is offered. PE is less well tolerated and is primarily used as a third choice and only for a few weeks to months to induce initial improvement. Once symptoms are improving, the dose of corticosteroids or HIG should be tapered with the goal of eventual discontinuation depending on patient response. Patients who do not respond to initial therapy, experience adverse effects from the initial immunomodulating agent, or require chronic treatment can be treated with another first-line agent or one of several second-line agents.

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“…Bei einem Patienten kam es nach anfänglich langer Remission nach 5 Jahren zu einem Rückfall [ 48 ] . In einer weiteren kleinen Serie haben 2 von 3 Patienten profi tiert [ 49 ] und in einem weiteren Fall konnte ein Patient alle weiteren Immuntherapien beenden [ 50 ] .…”

Section: Stammzelltransplantationunclassified

Neue Entwicklungen bei CIDP und MMN

Stangel

Kieseier

2011

Akt Neurol

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Zusammenfassung Chronisch immunvermittelte Polyneuropathien, wie die CIDP [chronisch inflammatorische (entz?ndliche) demyelinisierende Polyneuropathie] und die multifokal motorische Neuropathie (MMN), stellen im klinischen Alltag eine diagnostische und therapeutische Herausforderung dar. Die Heterogenit?t in der klinischen Pr?sentation als auch die nur wenigen klinischen Therapiestudien stellen Limitationen im Umgang mit diesen Krankheitsbildern dar. In den letzten Jahren wurden auf diesem Gebiet jedoch Fortschritte erzielt: Einerseits gibt es neue diagnostische Kriterien und Leitf?den, die im klinischen Alltag die differenzialdiagnostische Einordnung erleichtern, andererseits gibt es f?r die CIDP inzwischen eine klinische Studie h?chster Evidenzklasse, die sowohl die Kurz- als auch Langzeitwirksamkeit von intraven?sen Immunglobulinen demons?triert. In dem folgenden ?bersichtsartikel werden die rezenten diagnostischen und therapeutischen Erkenntnisse f?r die CIDP als auch die MMN zusammengestellt.

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Nonmyeloablative Autologous Hematopoietic Stem Cell Transplantation for Refractory Cidp

Cited by 46 publications

References 0 publications

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Haematopoietic stem cell transplantation in autoimmune diseases: From basic science to clinical practice

Inflammatory Demyelinating Neuropathies

Neue Entwicklungen bei CIDP und MMN

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