Progress in Hematopoietic Stem Cell Transplantation for CIDP

Qin, Zhen; Huang, Qiangru; Zou, Jiangying; Tang, Luosheng; Hu, Zhiping; Tang, Xiangqi

doi:10.7150/ijms.38363

Cited by 7 publications

(7 citation statements)

References 66 publications

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“…Although most patients have been effectively treated with immunosuppressive medications, high doses of steroids may not be able to alter the rapidly progressing clinical course of BCS. Other treatment options include plasma exchange and immunosuppressive medication, followed by autologous stem cell transplantation 21 …”

Section: Discussionmentioning

confidence: 99%

A rare case of Balo's disease in a young adult: A clinical presentation and management

Singh,

Almusleh,

Mandru

et al. 2024

Clinical Case Reports

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Key Clinical MessageAccurate diagnosis of rare neurological conditions like Balo's concentric sclerosis (BCS) is challenging but crucial for tailored treatment. Interdisciplinary collaboration and further research are essential to advance our understanding.AbstractThis case report presents a 32‐year‐old female patient with a puzzling neurological condition characterized by feverish feelings, joint pain, unclear speech, and an unsteady gait. Initial management did not lead to improvement, and further examination revealed neurological involvement and joint tenderness without signs of inflammatory arthritis. Laboratory investigations ruled out infectious and autoimmune causes. Magnetic resonance imaging (MRI) showed well‐defined lesions with concentric rings, leading to a diagnosis of Balo' concentric sclerosis. Treatment with intravenous methylprednisolone resulted in limited improvement. This case highlights the importance of thorough evaluation and collaboration in diagnosing rare neurological conditions. Further research is needed to enhance the understanding and treatment of rare neurological disorders.

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Section: Discussionmentioning

confidence: 99%

A rare case of Balo's disease in a young adult: A clinical presentation and management

Singh,

Almusleh,

Mandru

et al. 2024

Clinical Case Reports

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“…Hematopoietic stem cell transplantation was recommended for such refractory patients. HSCT involves eliminating or depleting a broad range of lymphoid and myeloid cells, including adaptive immune cells (T cells and B cells) and innate immune cells (natural killer cells, dendritic cells, monocytes and granulocytes), to rebuild a self-tolerant immune system and block recognition of autoantigenic epitopes [15,16,59,60,61], potentially leading to long-term, asymptomatic remission in refractory autoimmune diseases including refractory CIDP [30,43,62].…”

Section: Discussionmentioning

confidence: 99%

“…Hematopoietic stem cell transplantation is generally considered safe and feasible, although it is associated with both early and late adverse events [30,66]. Early complications, which often occur within 100 days of transplantation, may include febrile neutropenia, hypophosphatemia, infection, hypokalemia and transient dyspnea.…”

Section: F I G U R Ementioning

confidence: 99%

“…There has been evidence to suggest that HSCT may be effective in treating CIDP, particularly in severe, refractory cases. However, the available data on the use of HSCT in CIDP are contradictory and there is limited literature on its efficacy and safety in this patient population [29‐31]. In this review, the aim was to evaluate the potential therapeutic effects, benefits and complications of HSCT in the treatment of refractory CIDP patients.…”

Section: Introductionmentioning

confidence: 99%

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Efficacy of hematopoietic stem cell transplantation treatment in refractory chronic inflammatory demyelinating polyradiculoneuropathy: a systematic review and meta‐analysis

Zheng

Sun

et al. 2023

Euro J of Neurology

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Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare immune-mediated disorder that causes demyelination and axonal damage of the peripheral nerves. It is characterized by symmetrical weakness in the proximal or distal limbs, loss of sensation and loss of tendon reflexes [1-3]. The first-line therapies for CIDP are intravenous immunoglobulin (IVIG), therapeutic plasmapheresis (TPE) and corticosteroids [4,5]. However, approximately 20%-30% of patients with CIDP do not respond well to these therapies, and around 15% of patients remain refractory to all treatment options [6-10]. These patients often experience continued progression of their disease in the form of relapses or progressive disability, despite treatment efforts. The lack of response to treatment can have a significant physical and psychosocial impact on patients, highlighting the need for effective therapies to address the unmet needs of those refractory CIDP patients [11,12]. Hematopoietic stem cell transplantation (HSCT) is a widely accepted treatment for hematological cancers, other neoplastic disorders and severe immune deficiencies [13,14]. It is a major procedure that usually involves administering high-dose chemotherapy and immunodepleting serotherapy, typically anti-thymocyte

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“…HSCT is being utilized increasingly in severe refractory autoimmune disorders [ 213 ]. Evidence has been accumulating in favour of HSCT in cases of refractory MG with all patients achieving complete stable remission, although the benefits may not be permanent as evident in some autoimmune disorders such as chronic inflammatory demyelinating polyneuropathy [ 214 – 218 ]. Unfortunately, the phase I study of HSCT in refractory MG was terminated prematurely because of failure of recruitment and no observations have been published.…”

Section: Newer Biological Treatmentsmentioning

confidence: 99%

Pharmacotherapy of Generalized Myasthenia Gravis with Special Emphasis on Newer Biologicals

Menon

Bril

2022

Drugs

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Myasthenia gravis (MG) is a chronic, fluctuating, antibody-mediated autoimmune disorder directed against the post-synaptic neuromuscular junctions of skeletal muscles, resulting in a wide spectrum of manifestations ranging from mild to potentially fatal. Given its unique natural course, designing an ideal trial design for MG has been wrought with difficulties and evidence in favour of several of the conventional agents is weak as per current standards. Despite this, acetylcholinesterases and corticosteroids have remained the cornerstones of treatment for several decades with intravenous immunoglobulins (IVIG) and therapeutic plasma exchange (PLEX) offering rapid treatment response, especially in crises. However, the treatment of MG entails long-term immunosuppression and conventional agents are viable options but take longer to act and have a number of class-specific adverse effects. Advances in immunology, translational medicine and drug development have seen the emergence of several newer biological agents which offer selective, target-specific immunotherapy with fewer side effects and rapid onset of action. Eculizumab is one of the newer agents that belong to the class of complement inhibitors and has been approved for the treatment of refractory general MG. Zilucoplan and ravulizumab are other agents in this group in clinical trials. Neisseria meningitis is a concern with all complement inhibitors, mandating vaccination. Neonatal Fc receptor (FcRn) inhibitors prevent immunoglobulin recycling and cause rapid reduction in antibody levels. Efgartigimod is an FcRn inhibitor recently approved for MG treatment, and rozanolixizumab, nipocalimab and batoclimab are other agents in clinical trial development. Although lacking high quality evidence from randomized clinical trials, clinical experience with the use of anti-CD20 rituximab has led to its use in refractory MG. Among novel targets, interleukin 6 (IL6) inhibitors such as satralizumab are promising and currently undergoing evaluation. Cutting-edge therapies include genetically modifying T cells to recognise chimeric antigen receptors (CAR) and chimeric autoantibody receptors (CAAR). These may offer sustained and long-term remissions, but are still in very early stages of evaluation. Hematopoietic stem cell transplantation (HSCT) allows immune resetting and offers sustained remission, but the induction regimens often involve serious systemic toxicity. While MG treatment is moving beyond conventional agents towards target-specific biologicals, lack of knowledge as to the initiation, maintenance, switching, tapering and long-term safety profile necessitates further research. These concerns and the high financial burden of novel agents may hamper widespread clinical use in the near future.

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Progress in Hematopoietic Stem Cell Transplantation for CIDP

Cited by 7 publications

References 66 publications

A rare case of Balo's disease in a young adult: A clinical presentation and management

A rare case of Balo's disease in a young adult: A clinical presentation and management

Efficacy of hematopoietic stem cell transplantation treatment in refractory chronic inflammatory demyelinating polyradiculoneuropathy: a systematic review and meta‐analysis

Pharmacotherapy of Generalized Myasthenia Gravis with Special Emphasis on Newer Biologicals

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