Background: Stiff person syndrome spectrum disorders (SPSD) are a rare group of disabling neuroimmunological disorders. SPSD often requires immune therapies, especially in the setting of inadequate response to symptomatic treatments. The safety and efficacy of therapeutic plasma exchange (TPE) in SPSD remains uncertain. Objectives: To describe the safety, tolerability, and efficacy of TPE in patients with SPSD. Design: A retrospective observational study. Methods: A retrospective review of SPSD patients seen at Johns Hopkins Hospital (JHH) from 1997 to 2021 was performed. Patient demographics/history, examination/diagnostic findings, treatment response, and TPE-related complications were recorded. Assessment for any associations between clinical characteristics, including age, sex, clinical phenotype, and time on immunotherapy, and response to TPE 3 months after treatment was performed. A subgroup of 18 patients treated with TPE at JHH and 6 patients treated with TPE at outside institutions were evaluated for any change in usage of symptomatic medications 3 months after the TPE treatment. Literature review of SPSD and TPE was also conducted. Results: Thirty-nine SPSD patients were treated with TPE (21 at JHH and 18 at outside institutions); median age 48 years, 77% female, median modified Rankin Scale 3; mean initial anti-GAD65 antibody titer was 23,508 U/mL. Twenty-four patients (62%) had classic SPS, 10 (26%) had SPS-plus, 2 (5%) had progressive encephalomyelitis with rigidity and myoclonus, and 3 (8%) had pure cerebellar ataxia. All patients were on symptomatic treatments, 30 (77%) previously received IVIg, and 3 (8%) previously received rituximab. Four patients (10%) had a TPE-related adverse event. One developed asymptomatic hypotension, another had both line thrombosis and infection, and two had non-life-threatening bleeding events. Twenty-three (59%) patients reported improvement in symptoms after TPE. Of the subgroup of 24 patients evaluated for any change in usage of symptomatic medications 3 months after the TPE treatment, 14 (58%) required fewer GABAergic symptomatic medications. Literature review identified 57 additional patients with SPSD; 43 (75%) reported temporary improvement after TPE. Conclusion: The majority of patients treated with TPE had improvement. Moreover, most patients evaluated for any change in usage of symptomatic medications after the TPE treatment no longer required as much symptomatic medications months after TPE. TPE appears safe and well-tolerated in SPSD. Further studies are needed to assess the long-term efficacy of TPE in SPSD and identify which patients may benefit the most from TPE.
Background Prior to laboratory‐based blood donor screening for Babesia, transfusion‐transmitted babesiosis (TTB) was a leading infectious risk to the blood supply in the United States. Case Report A 30‐year‐old man with sickle cell disease (SCD) who had been on a chronic automated red cell exchange (RCE) regimen since childhood, presented approximately 2 months after an RCE, with fever, neck pain, and photophobia. Meningitis was excluded, and he was discharged. He presented again 2 days later with persistent fever, chills, headache, fatigue, and loss of appetite. Results On examination, the patient was febrile but hemodynamically stable. Intra‐erythrocytic inclusions were identified on a peripheral blood smear (<0.5%). B. microti IgM and IgG titers were >1:320 (Reference <1:20) >1:1024 (Reference <1:64), respectively. B. microti was confirmed by nucleic acid testing. The patient lived in a Babesia endemic state but had no risk factors for tick‐borne acquisition. Of the 65 units he received in the preceding 6 months, 58 had been screened for Babesia. One of the donors of the 7 untested units was B. microti seropositive (titer 1:128; Reference 1: 64). The donor was asymptomatic and resided in a state in which Babesia screening was not required. He reported traveling in the year before his donation. Conclusion Although rare, TTB is still possible despite regional screening, underscoring the need for provider vigilance and education, especially in non‐endemic areas. Patients with SCD are particularly vulnerable given their high frequency of transfusion and complex needs requiring blood procurement from states where Babesia screening is not mandatory.
ObjectiveTo describe the safety, tolerability, and response to TPE in patients with SPSD.BackgroundStiff person syndrome spectrum disorders (SPSD) are a rare group of disabling neuroimmunological disorders. SPSD often require immune therapies especially in the setting of inadequate response to symptomatic treatments. The safety and efficacy of therapeutic plasma exchange (TPE) in SPSD is unclear.Design/MethodsA retrospective review of medical records for patients with SPSD seen at Johns Hopkins Hospital was performed. Patient characteristics, exam findings, diagnostic studies, treatment response and TPE-related complications were recorded. We also conducted linear regression models to assess for possible predictive factors for TPE response. Literature review of SPSD and TPE was also conducted.ResultsThirty-six SPSD patients were treated with TPE; mean age was 48 years, 81% female, and average anti-GAD65 antibody titer was 42352 U/mL (range, 0-309,902). Twenty-two patients had classic SPS, 10 had SPS-plus, and 3 had other phenotypes. Thirty-three patients were treated for acute exacerbations, and 3 were on maintenance TPE. There were 4 (11.1%) TPE-related complications (catheter infection, catheter thrombosis, hemorrhage), but no deaths or anaphylaxis. Twenty patients (55.6%) reported improvement in symptoms after TPE, 13 reported no change, and 3 reported worsening of symptoms. Of the 36 total patients who received TPE, 21 received TPE at Johns Hopkins Hospital for an acute exacerbation of their condition, with 12 requiring fewer anti-spasmodic medications 3 months after TPE treatment. There were no predictive factors in a positive treatment response to TPE. Literature review identified 42 more patients; 69% of these patients reported a temporary improvement in their condition.ConclusionsWe describe the safety and tolerability of TPE in patients with SPSD and show that TPE-related complications are uncommon and manageable. Additionally, many patients with SPSD derived improvement with TPE. Further studies could help inform clinicians when to use TPE in SPSD.
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