Autologous Hematopoietic Stem Cell Transplantation for Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Abstract

Background: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) refractory to conventional therapy can lead to marked disability and represents a therapeutic challenge. Objective: To report five cases of treatment-refractory disabling CIDP treated with autologous hematopoietic stem cell transplantation (AHSCT). Methods: This was a retrospective cohort study from a tertiary care referral center for both neuromuscular disease and AHSCT. Patients with CIDP treated with AHSCT between 2008 and 2020 were included. All patients had major persistent and disabling neuropathic deficits despite combinations of intensive immunosuppressive therapy. The primary outcome measures were: Medical Research Council sum score, Overall Neuropathy Limitations Scale and requirement for ongoing CIDP immunotherapy after transplantation. We also analyzed safety outcomes by documenting all severe AHSCT-related complications. Results: Five patients with refractory CIDP underwent AHSCT. Three were classified as manifesting a typical syndrome, two were classified as the multifocal Lewis Sumner variant. The mean age at time of CIDP diagnosis was 33.4 years (range 24–46 years), with a median delay of 46 months (range 21–135 months) between diagnosis and AHSCT. The median follow-up period was 41 months. All five patients were able to wean off CIDP-related immunotherapy. Marked improvements in Medical Research Council scale and overall Neuropathy Limitations Scale were noted in 4/5 patients. One patient with longstanding neurogenic atrophy showed no improvement in disability scales. There were no treatment-related deaths or critical illnesses. Conclusions: AHSCT can achieve marked sustained clinical improvement of refractory CIDP and may allow for weaning off long-term complex immunotherapies.

Stem Cell Therapy in Neuroimmunological Diseases and Its Potential Neuroimmunological Complications

“…A total of 20 case reports and one phase I/II study investigating stem-cell therapies in 108 patients (37 women, age 17–75 years, disease duration 1–38 years, follow-up 7–78 months) suffering from CIDP, MMN, and myasthenia gravis were found [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. Of these patients, 95 suffered from CIDP, 10 suffered from myasthenia gravis, and one each suffered from MMN, myasthenia gravis and concomitant amyotrophic lateral sclerosis (ALS), and myasthenia gravis and concomitant polymyositis [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ].…”

“…A total of 20 case reports and one phase I/II study investigating stem-cell therapies in 108 patients (37 women, age 17–75 years, disease duration 1–38 years, follow-up 7–78 months) suffering from CIDP, MMN, and myasthenia gravis were found [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. Of these patients, 95 suffered from CIDP, 10 suffered from myasthenia gravis, and one each suffered from MMN, myasthenia gravis and concomitant amyotrophic lateral sclerosis (ALS), and myasthenia gravis and concomitant polymyositis [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. Due to consideration of several case reports, the patients’ symptoms and disease-related disability were highly heterogeneous and scored with different clinical evaluation tools (modified Rankin scale, inflammatory neuropathy cause and treatment score, Rasch-built overall disability scale, medical research council sum score, myasthenia gravis foundation of America score, and myasthenia gravis composite score) [ 104 , 1...…”

“…Of these patients, 95 suffered from CIDP, 10 suffered from myasthenia gravis, and one each suffered from MMN, myasthenia gravis and concomitant amyotrophic lateral sclerosis (ALS), and myasthenia gravis and concomitant polymyositis [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. Due to consideration of several case reports, the patients’ symptoms and disease-related disability were highly heterogeneous and scored with different clinical evaluation tools (modified Rankin scale, inflammatory neuropathy cause and treatment score, Rasch-built overall disability scale, medical research council sum score, myasthenia gravis foundation of America score, and myasthenia gravis composite score) [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. On the one hand, there were patients, who were treated with stem-cell therapies due to the dependence of regular treatment (IVIG and plasmapheresis), but who responded well to this initial treatment and recovered completely [ 104 , 105 , ...…”

“…Due to consideration of several case reports, the patients’ symptoms and disease-related disability were highly heterogeneous and scored with different clinical evaluation tools (modified Rankin scale, inflammatory neuropathy cause and treatment score, Rasch-built overall disability scale, medical research council sum score, myasthenia gravis foundation of America score, and myasthenia gravis composite score) [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. On the one hand, there were patients, who were treated with stem-cell therapies due to the dependence of regular treatment (IVIG and plasmapheresis), but who responded well to this initial treatment and recovered completely [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. These patients were not severely disabled, and symptoms mostly did not influence daily activities [ 104 , 105 , 106 , 107 …”

“…On the one hand, there were patients, who were treated with stem-cell therapies due to the dependence of regular treatment (IVIG and plasmapheresis), but who responded well to this initial treatment and recovered completely [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. These patients were not severely disabled, and symptoms mostly did not influence daily activities [ 104 , 105 , 106 , 107 , 108 , 109 , 110 , 111 , 112 , 113 , 114 , 115 , 116 , 117 , 118 , 119 , 120 , 121 , 122 , 123 , 124 ]. On the other hand, there were patients, who were treatment-refractory and/or highly disabled (including the need for intensive care and ventilation) [ 104 , 105 , 106 , 107 , 108 , 109 , 110 ,…”