Subcutaneous Immunoglobulin Treatment Research Articles

Facilitated Subcutaneous Immunoglobulin Treatment Increases the Quality of Life and Decreases the Number of Infections and Hospitalizations in Children with Primary Immunodeficiencies

Introduction: Immunoglobulin replacement therapy is an effective lifelong treatment modality used in patients with primary immunodeficiency to prevent and/or reduce the incidence of serious infections. Facilitated subcutaneous immunoglobulin (fSCIG) was developed to combine the advantages of intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) and is the latest method of immunoglobulin G (IgG) administration. In this study, switching to fSCIG administration in primary immunodeficiency patients receiving regular IVIG or SCIG therapy was evaluated, and serum IgG trough levels, frequency of infections, frequency and duration of hospitalizations, duration of absence from school/work, and quality of life were determined. Methods: In this study, fifteen patients with primary immunodeficiency who were previously receiving IVIG or SCIG treatment, followed by fSCIG, were evaluated retrospectively. Age, diagnosis, current complications, mean IgG value, frequency of infection, frequency of hospitalization, and duration of absenteeism from school and work were recorded during and before fSCIG treatment. At the beginning of fSCIG treatment, at 6th and 12th months, “The Quality of Life Scale” was also evaluated in patients and parents. Results: The most common indications for initiation of fSCIG treatment were the difficulty of access to the hospital and the long transfusion periods. No systemic adverse reactions were reported except for redness, swelling, and mild pain on the injection site. The median IgG values for the last 1 year were 529.6 mg/dL for IVIG (n = 9), 876.2 mg/dL for SCIG (n = 6) and 856.7 mg/dL for fSCIG (n = 15, all patients) treatment. The frequency of infections and the number of hospitalizations decreased significantly in the fSCIG group compared to both previous treatment modalities. There was a significant increase in the quality of life score of the patients and their families when compared with previous treatment modalities. Conclusion: fSCIG is an effective treatment method and is well tolerated in patients with immunodeficiency. It provides stable immunoglobulin levels and excellent protection against infections and offers the patients the possibility of home-based therapy.

Subcutaneous immunoglobulin treatment preferences among patients with chronic inflammatory demyelinating polyradiculoneuropathy: A discrete choice experiment

Subcutaneous immunoglobulin treatment preferences among patients with chronic inflammatory demyelinating polyradiculoneuropathy: A discrete choice experiment

Experience with the use of Hizentra, an immunoglobulin preparation for subcutaneous administration, in patients with primary immunodeficiency diseases

Immunoglobulin replacement therapy is the gold standard of treatment for patients with antibody deficiencies. We aimed to investigate the efficacy and safety of replacement therapy with subcutaneous immunoglobulin (SCIG) Hizentra in patients with primary immunodeficiencies. This study was approved by the Independent Ethics Committee and the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. All patients and / or their legal representatives gave informed consent for this treatment. In our study, 12 patients under 18 years of age with various forms of primary immunodeficiencies who had previously received intravenous immunoglobulin were switched to SCIG treatment to receive weekly infusions of Hizentra. Therapy SCIG was administered weekly at a dose of 0.1–0.15 g/kg by rapid push infusion. All patients received Hizentra for at least 3 months. None of the patients included in the study developed severe infections. Immunoglobulin G levels in blood after 3 months of SCIG therapy were significantly higher compared to those achieved on previous intravenous immunoglobulin therapy. There were no severe adverse events associated with Hizentra administration. Our study demonstrated Hizentra to be effective and safe for the treatment of children with various forms of primary immunodeficiencies.

Facilitated Subcutaneous Immunoglobulin Treatment in Patients with Immunodeficiencies: the FIGARO Study

PurposeThe FIGARO study aims to provide insights on real-world utilization and tolerability of facilitated subcutaneous immunoglobulin (fSCIG) for primary immunodeficiency disease (PID) or secondary immunodeficiency disease (SID).MethodsThis prospective, multicenter, observational study, evaluated medical records, charts, and diaries of patients who had received at least 1 fSCIG infusion for PID or SID. Data were analyzed by cohort (PID, SID) and age groups (pediatric [< 18 years], adult [18–64 years], older adult [≥ 65 years]). Patients were followed up to 36 months.ResultsThe study enrolled 156 patients: 15 pediatric, 120 adult, 21 older-adult. Twelve-month follow-up data were available for 128 patients. fSCIG was mainly prescribed for PID among patients aged < 65 years and for SID among older adults. At inclusion, 75.6% received their fSCIG infusion at home, and 78.7% self-administered. Adults were more likely to receive their initial infusion at home and self-administer (81.7% and 86.6%, respectively) than pediatric patients (53.3% each) and older adults (57.1% and 52.4%, respectively). At 12 months, the proportion of patients infusing at home and self-administering increased to 85.8% and 88.2%. Regardless of age, most patients self-administered the full fSCIG dose at home every 3–4 weeks and required a single infusion site. The tolerability profile was consistent with previous pivotal trials. Acute severe bacterial infections occurred in 0%–9.1% of patients during follow-up visits (full cohort).ConclusionsFIGARO confirms the feasibility, tolerability, and good infection control of fSCIG in PID and SID patients across the age spectrum in both the home-setting and medical facility.Trial registration numberClinicalTrials.gov NCT03054181

Real-World Evidence of Tolerability of 20% Subcutaneous Immunoglobulin Treatment

PurposeThe safety and efficacy of subcutaneous immune globulin 20% (human) solution (Ig20Gly) were demonstrated in clinical trials. However, real-world evidence of the tolerability of self-administered Ig20Gly in elderly patients is lacking. We describe real-world patterns of Ig20Gly usage for 12 months in patients with primary immunodeficiency diseases (PIDD) in the USA.MethodsThis retrospective chart review of longitudinal data from 2 centers included patients aged ≥ 2 years with PIDD. Ig20Gly administration parameters, tolerability, and usage patterns were assessed at initial and subsequent 6- and 12-month infusions.ResultsOf 47 enrolled patients, 30 (63.8%) received immunoglobulin replacement therapy (IGRT) within 12 months before starting Ig20Gly, and 17 (36.2%) started IGRT de novo. Patients were predominantly White (89.1%), female (85.1%), and elderly (aged > 65 years, 68.1%; median age = 71.0 years). Most adults received at-home treatment during the study, and most self-administered at 6 months (90.0%) and 12 months (88.2%). Across all time points, infusions were administered at a mean rate of 60–90 mL/h/infusion, using a mean of 2 sites per infusion, on a weekly or biweekly frequency. No emergency department visits occurred, and hospital visits were rare (n = 1). Forty-six adverse drug reactions occurred in 36.4% of adults, mostly localized site reactions; none of these or any adverse events led to treatment discontinuation.ConclusionThese findings demonstrate tolerability and successful self-administration of Ig20Gly in PIDD, including elderly patients and patients starting IGRT de novo.

Continuous increase of immunoglobulin therapy in Iceland

• Immunoglobulin therapy is a beneficial treatment for a variety of registered and off-label • indications and use of immunoglobulin treatment has been continually increasing. We confirmed the increasing use of immunoglobulin in Iceland and conclude that continually increasing demand and global shortage dictates careful consideration of indications before initiating treatment with immunoglobulins. The use of immunoglobulin treatment has been increasing for various indications. The objective of this study was to evaluate clinical use of immunoglobulin therapy in Iceland, estimate off-label usage and describe possible changes in this treatment option. All prescriptions for intravenous or subcutaneous immunoglobulin treatment require authorisation from Landspitali University Hospital. Patient information is registered and was retrieved for the period 2010 to 2019. A total of 921 patients received immunoglobulin treatment in the study period, intravenous therapy was given in 895 (97.2%) of the cases. Registered indications were 667, off-label indications were 245 and uncertain were nine. The total annual number of patients receiving immunoglobulin treatment increased from 87 in 2010 to 392 in 2019. The increase in immunoglobulin usage was both for registered and off-label indications, 61 to 309 and 26 to 83, respectively. Continually increasing demand and global shortage dictates careful consideration of indications before initiating or continuing treatment with immunoglobulins. With global shortage of immunoglobulins, careful consideration and patient selection is important. Increasing immunoglobulin usage improves treatment for many patients. However, immunoglobulins are a limited resource and prudent use is imperative.

Cutaquig® Is Well Tolerated in Immunodeficient Patients Who Did Not Tolerate Other Subcutaneous Immunoglobulin Products.

Subcutaneous immunoglobulin (SCIG) treatment is generally tolerable, but some patients may experience adverse events to one or more SCIG products. We investigated whether 16.5% Cutaquig® treatment offered a tolerable and safe alternative treatment for immunodeficient patients. A one-year prospective cohort study was conducted at a single center in Ottawa, Canada. Adult immunodeficient patients who reported previous intolerability, adverse events, or other difficulty to other 20% SCIG product(s) were recruited to start on 16.5% Cutaquig®. Treatment tolerability, safety, and quality of life were observed and described. Seven out of ten patients tolerated Cutaquig®. There were no serious or severe adverse events related to the treatment. Three moderate infections were reported (two urinary tract infections and one injection site infection). The mean serum IgG level at the end of the study was comparable to baseline levels recorded before the study: 9.6 ± 4.5 vs. 7.6 ± 4.3 g/L, p = 0.07. The overall health and health domain changes in the SF-36 and quality of life tests using the EQ visual analog scale improved by 21.5% (p = 0.38), 16.7% (p = 0.29), and 7.7% (p = 0.23), respectively. Cutaquig® may be used as an alternative treatment option for patients who did not tolerate 20% SCIG products.

Optimizing chronic inflammatory demyelinating polyneuropathy care with subcutaneous immunoglobulin: The Polyneuropathy and Treatment with Hizentra Open-Label Extension (PATH OLE) study and beyond

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a heterogeneous, acquired autoimmune neurological disorder affecting peripheral nerves. CIDP is characterized by progressive weakness, reduced or absent tendon reflexes and impaired sensory function in the lower and upper limbs. CIDP diagnosis is mainly based on clinical, laboratory and electrophysiologic criteria and there are currently no diagnostic or prognostic biomarkers. First-line treatment options include corticosteroids, intravenous immunoglobulin (IVIg) and plasma exchange (PLEX). While IVIg and corticosteroids are the most common therapies administered for CIDP, there are challenges associated with their use, including systemic adverse events (AEs), some of which can be serious. Studies have shown that subcutaneous immunoglobulin (SCIg) may be associated with improved quality of life, which is attributed partially to the patients’ freedom to administer SCIg at home and at their convenience. While AEs with SCIg mostly consist of local site reactions, SCIg is associated with fewer systemic AEs compared with IVIg, and these are commonly mild, though severe reactions may rarely occur. A number of studies in the last decade have assessed SCIg in CIDP. One of these studies, the Polyneuropathy and Treatment with Hizentra® (PATH) study, was a global phase 3, double-blind, randomized, placebo-controlled trial that assessed the efficacy, safety, and tolerability of SCIg treatment in patients with CIDP. Based on the results of the PATH study, the US Food and Drug Administration (FDA) approved SCIg as a maintenance treatment for CIDP in 2018. This review summarizes and discusses the results of the PATH study and its open-label extension (OLE) study and provides an overview of the April 2021 update to the Hizentra® FDA-approved U.S. package insert based on findings from the PATH OLE. In addition, the review highlights key elements of the second revision of the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) guideline for the diagnosis and treatment of CIDP. Finally, this review discusses the characteristics of patients with CIDP who may benefit from SCIg treatment.

Long-termsafety of facilitated subcutaneous immunoglobulin treatment in pregnant women with primary immunodeficiency diseases: results from a registry study.

Aim: Clinical outcomes of women who become pregnant during/after facilitated subcutaneous immunoglobulin (fSCIG) treatment are not well characterized. Materials & methods: This noninterventional, prospective, open-label, postauthorization, pregnancy registry study assessed safety outcomes in mothers with primary immunodeficiency diseases who had ever received fSCIG before/during pregnancy and their infants (n=7). Enrolled women received alternative treatment (arm 1: n=2) or continued fSCIG (arm 2: n=7) during pregnancy. Results: No treatment-related adverse events (AEs)/serious AEs (SAEs) were reported. 13AEs occurred in mothers, including two SAEs (thrombocytopenia, pre-eclampsia; arm 2). A total of 17AEs occurred in infants, including two SAEs (cleft lip, talipes calcaneovalgus; arm 2) with normal growth/development. Conclusion: Findings provide limited but useful safety data regarding women who received fSCIG before/during pregnancy and the growth/development of their infants. Clinical Trial registration: NCT02556775 (ClinicalTrials.gov); EUPAS5798.

Replacement and Immunomodulatory Activities of 20% Subcutaneous Immunoglobulin Treatment: A Single-Center Retrospective Study in Autoimmune Myositis and CVID Patients.

BackgroundImmunoglobulin (Ig) replacement therapy represents a life-saving treatment in primary antibody deficiencies. The introduction of subcutaneous Ig (SCIg) administration brings a major improvement in quality of life for patients, compared to the traditional intravenous administration. In recent years, an additional role has been proposed for Ig therapy for various inflammatory and immune-mediated diseases. Consequently, the use of SCIg has expanded from immunodeficiencies to immune-mediated diseases, such as polymyositis (PM) and dermatomyositis (DM). Given the rarity of these conditions, it is still difficult to evaluate the real impact of SCIg treatment on PM and DM, and additional data are constantly required on this topic, particularly for long-term treatments in real-life settings.AimThis study aimed to increase the knowledge about the anti-inflammatory and immunomodulatory effects of SCIg treatment for myositis. To this aim, a long-term evaluation of the effectiveness of 20% human SCIg treatment (20% SCIg, Hizentra®, CSL Behring) was carried out in patients with PM/DM in care at our Center. In addition, an evaluation of the 20% SCIg therapy in CVID patients was provided. This analysis, beside adding knowledge about the use of SCIg therapy in this real-life setting, was intended as a term of comparison, regarding the safety profile.ResultsResults support the beneficial effect and tolerability of long-term 20% SCIg therapy in PM/DM patients, reporting a significant improvement in creatine kinase levels, muscle strength, skin conditions, dysphagia, disease activity (MITAX score) and disability (HAQ-DI score). None of the patients reported systemic reactions. The duration of the reported local reactions was a few hours in 80% of the patients, and all resolved spontaneously. CVID patients reported an improvement in all the considered effectiveness parameters at the end of 20% SCIg therapy. The frequency of the adverse events reported by PM/DM patients was not different from what reported in CVID patients, where the use of SCIg therapy is more consolidatedConclusionsThis study suggests that 20% SCIg treatment represents a viable and safe treatment for PM/DM patients and a valid therapeutic alternative to IVIg, with important advantages for patients’ quality of life.

Subcutaneous immunoglobulin treatment for chronic inflammatory demyelinating polyneuropathy.

Immunoglobulin G (IgG) therapy is an established long‐term treatment in chronic inflammatory demyelinating polyneuropathy (CIDP) that is commonly administered intravenously (IVIg). The subcutaneous immunoglobulin (SCIg) administration route is a safe and effective alternative option, approved by the United States Food and Drug Administration (FDA) in 2018, for maintenance treatment of adults with CIDP. Physicians and patients alike need to be aware of all their treatment options in order to make informed decisions and plan long‐term treatment strategies. In this review, we collate the evidence for SCIg in CIDP from all published studies and discuss their implications and translation to clinical practice. We also provide guidance on the practicalities of how and when to transition patients from IVIg to SCIg and ongoing patient support. Evidence suggests that IVIg and SCIg have comparable long‐term efficacy in CIDP. However, SCIg can provide additional benefits for some patients, including no requirement for venous access or premedication, and reduced frequency of systemic adverse events. Local‐site reactions are more common with SCIg than IVIg, but these are mostly well‐tolerated and abate with subsequent infusions. Data suggest that many patients prefer SCIg following transition from IVIg. SCIg preference may be a result of the independence and flexibility associated with self‐infusion, whereas IVIg preference may be a result of familiarity and reliance on a healthcare professional for infusions. In practice, individualizing maintenance dosing based on disease behavior and determining the minimally effective IgG dose for individuals are key considerations irrespective of the administration route chosen.

Efficacy and quality of life assessment in the use of subcutaneous immunoglobulin treatment for children with primary immunodeficiency disorder.

Introduction. Most patients with primary and secondary immunodeficiencies need regular Intravenous Immunoglobulin (IVIG) or Subcutaneous Immunoglobulin (SCIG) treatment. This study aimed to evaluate the serum IgG trough levels, frequency of mild and severe infections, frequency and duration of hospitalization, duration of absence of school, and quality of life in patients switching their IVIG therapy to SCIG administration. Materials. Twenty-nine patients with immunodeficiency on regular IVIG treatment and who agreed to receive SCIG treatment were included. Seven patients discontinued treatment after the first SCIG administration. We collected data regarding serum IgG levels, annual numbers of infections, hospital admissions, and adverse events prior to and following SCIG initiation. PedsQL tests such as Scale Total Score (STS), Physical Health Total Score (PHTS), Psychosocial Health Total Score (PsyHTS), emotional functionality, social functionality, school/work problems score were calculated separately for all patients and their parents. Results. In twenty-two cases who were diagnosed as primary immunodeficiency, the most common indication for initiation of SCIG treatment was the long transfusion period of IVIG treatments and the difficulty of access to the hospital. No systemic side effects were noted except local redness, pain, and swelling on the injection site. The median IgG value was 588.9 mg/dl during IVIG treatment and 872 mg/dl one year after SCIG treatment. Annual frequency of infections and absence to school/work decreased significantly in the SCIG group while the annual number of hospitalizations and hospital stay time did not change significantly. There was a significant increase in the "quality of life" scores of the patients and their families. Conclusions. SCIG treatment provides ideal and protective immunoglobulin levels and offers the comfort of treatment in their home environment, thus increasing the patient's satisfaction and quality of life.

Long-term treatment with subcutaneous immunoglobulin in multifocal motor neuropathy

Multifocal motor neuropathy (MMN) is a rare disease with a prevalence of less than 1 per 100,000 people. Intravenous immunoglobulin (IVIG) therapy, performed for a long-term period, has been demonstrated able to improve the clinical picture of MMN patients, ameliorating motor symptoms and/or preventing disease progression. Treatment with subcutaneous immunoglobulin (SCIg) has been shown to be as effective as IVIG. However, previously published data showed that follow-up of MMN patients in treatment with SCIg lasted no more than 56 months. We report herein the results of a long-term SCIg treatment follow up (up to 96 months) in a group of 8 MMN patients (6 M; 2F), previously stabilized with IVIG therapy. Clinical follow-up included the administration of Medical Research Council (MRC) sum-score, the Overall Neuropathy Limitation Scale (ONLS) and the Life Quality Index questionnaire (LQI) at baseline and then every 6 months. Once converted to SCIg, patients’ responsiveness was quite good. Strength and motor functions remained stable or even improved during this long-term follow-up with benefits on walking capability, resistance to physical efforts and ability in hand fine movements.

Use of subcutaneous immunoglobulin in stiff person syndrome: Case series.

Introduction:Intravenous immunoglobulin (IVIG) has been shown to be effective for the treatment of stiff person syndrome (SPS). However, some patients might not tolerate it. We report the tolerability profile of subcutaneous immunoglobulin (SCIg) in patients with SPS who did not tolerate IVIG. To our knowledge, the use of SCIg in SPS has not been reported before in a case series.Patient concerns:The five patients included in this case series presented with various combinations of symptoms of spasms, axial and limb stiffness, and exaggerated responses to outside stimuli. These symptoms often lead to gait and functional impairment.Diagnosis:Patients were diagnosed with classic SPS as they met the clinical criteria, which require the presence of spasms, axial rigidity, and hyperexcitability.Interventions:Subcutaneous immunoglobulin infusion.Outcomes:Five patients were identified that were treated with SCIg. Three tested positive for serum anti-glutamic acid decarboxylase 65 antibodies prior to any treatment. The mean age at SCIg initiation was 33 years (range: 22–47). The mean duration of SPS prior to SCIg initiation was 5.9 years (range: 2.5–7). All patients used IVIG for at least two months (up to 18 months) but switched to SCIg due to IVIG side effects. Duration of SCIg use ranged from 4 months to 6 years (mean, 19.2 months). Upon switching to SCIg, the SPS symptoms remained stable. SCIg was well-tolerated in most as only one patient discontinued SCIg due to side effects.Conclusion:This case series highlights that SCIg could be a treatment option for patients with SPS, especially when IVIG is not feasible. Injection site reactions might be a limiting factor in some patients treated with SCIg. Prospective controlled studies are needed to confirm SCIg treatment durability and efficacy.

Long-term Safety of Facilitated Subcutaneous Immunoglobulin Treatment in Patients With Primary Immunodeficiency Diseases: Interim Analysis from a Post-authorization Safety Study

fSCIG (facilitated immune globulin infusion 10% [human] with recombinant human hyaluronidase [rHuPH20]) is a subcutaneously administered IG, approved for patients with primary immunodeficiency diseases (PIDD). The objective of this study was to acquire data from clinical practice on long-term safety of fSCIG in patients with PIDD.

Longitudinal study of intravenous versus subcutaneous immunoglobulin replacement therapy in hematological malignancy.

To present findings from a longitudinal study on infection risk, mortality, and patient perspective of intravenous immunoglobulin (IVIg) and subcutaneous immunoglobulin (SCIg) treatment for patients with hypogammaglobulinemia secondary to hematological malignancy or its treatment (abbreviated as SID). Observational study period included final year of IVIg (13 patients) and of the first 3 years of SCIg (17 patients) with SID. Data were collected on clinical outcomes from medical records and patient perception via study specific questionnaire. The median age was 63 years (53-76 years), and for 82.4% of patients their hematological malignancy was in complete remission. The annual mean serum IgG trough levels remained stable over the 4 years and were 7.0g/L (±2.77g/L) with IVIg, and 8.0g/L (±1.75g/L), 8.7g/L (±2.75g/L), and 7.6g/L (±2.89g/L) (year 1, 2, and 3, respectively) with SCIg. While the annual infection rate was similar, the rate of hospitalization due to infection fluctuated, with 37%, 9%, 15%, and 32% in year 1, 2, 3, and 4 respectively. There were no systemic adverse events with IVIg or SCIg. Patients reported a strong preference for SCIg. One patient died due to progression of underlying disease and infection within the study period. SCIg was the preferred treatment mode over IVIg in our cohort, but both were well tolerated without any systemic adverse events in 4-year follow up. The dosage and serum IgG levels were stable throughout. However, the number of infections requiring hospitalization fluctuated. It is anticipated that these findings encourage more hospitals to offer SCIg for SID patients.

Safety, Efficiency and Treatment Satisfaction in Children with Primary Immunodeficiency Receiving Subcutaneous Immunoglobulin Treatment

OBJECTIVEPatients with Inborn Errors of Immunity, also known as Primary Immunodeficiency (PID), are prone to recurrent bacterial infections and these patients often require lifelong IgG replacement therapy. The aim of this presentation is to evaluate the efficacy, safety, and patient satisfaction in PID patients receiving subcutaneous immunoglobulin (SCIG) treatment and to share our expe-riences.METHODSTwenty-one patients who were followed up with the diagnosis of PID by our Pediatric Allergy and Immunology Clinic and received regular intravenous immunoglobulin therapy (IVIG) befo-re starting SCIG treatment were included in the study.RESULTSA total of 21 patients were included in the study. 10 of the patients (47.6%) were female, 11 (52.4%) were male, and the mean age was 8.8±4.42 years. Five of the patients were Syrian patients living in the refugee camp. Threshold IgG levels of the patients were evaluated every 3 months. IgG levels were significantly higher than baseline IVIG levels at weeks 3, 6, and 12 of SCIG treatment, respectively. There was no significant difference between 3rd, 6th and 12th months of SCIG treatment. A statistically significant decrease was observed in the frequency of infections in patients who received SCIG treatment (p=0.003). During SCIG treatment, the total infection rate was 4.1/person/year. According to the TSQM-9 satisfaction questionnaire, the annual hospitalization rate was 0.9/patient/year for IVIG and 0.4/patient/year for SCIG (p>0.005), and 61.9% of patients were moderately satisfied, 14.2%. 19% were very satisfied and 4.7% were not satisfied with the treatment. When the satisfaction criteria were evaluated, it was observed that the patients mostly (71%) were satisfied with the absence of vascular access prob-lems and the comfort of self-application at home.CONCLUSIONSCIG therapy causes high serum IgG levels and a reduced frequency of infections and can be a safe, effective, and well-tolerated treatment alternative in patients with PID with high patient satisfaction.

Chronic immunoglobulin maintenance therapy in myasthenia gravis.

Long-term treatment of myasthenia gravis (MG) includes symptomatic and course-modifying therapies that target the immune system. Recently, both intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) have emerged as viable options for chronic therapy, considering the favourable safety-efficacy profile and possible immunosuppressant sparing properties. The aim was to investigate the outcomes of the long-term care of generalized MG with immunoglobulin (Ig). This is a retrospective, repeated-measures design study. Charts of generalized MG patients, treated with IVIG/SCIG for at least 6months, from January 2015 to January 2020, were analysed. The primary outcome was the mean change in Myasthenia Gravis Impairment Index (MGII) after treatment with Ig, comparing baseline to IVIG and SCIG treatment periods. Secondary outcomes included the changes in pyridostigmine, immunosuppressive medications and patient-reported outcome 'percentage of normal' (0%-100%). Thirty-four patients were treated with chronic Ig therapy (30 IVIG/SCIG, three SCIG, one IVIG). The mean durations of IVIG and SCIG periods were 21.8±19.4 (range 3-64) months and 19.5±11.3 (range 5-45) months respectively. There was a significant reduction in MGII scores (27.7±15.7 baseline; 22.0±17.4 IVIG period; 19.5±18.1 SCIG period; F=17.9; d.f.=1.7; P<0.01), pyridostigmine and immunosuppressant use (P=0.00). The outcome 'percentage of normal' had a significant positive association with both treatments (P=0.00). Our study results suggest that patients can be successfully transitioned to IVIG and from IVIG to SCIG in the chronic treatment of generalized MG with reductions in impairments and use of other medications and improvement in overall status with Ig therapy. Prospective, randomized studies are needed to clarify costs and comparative effectiveness.

Primer İmmün Yetmezlik Tanısıyla Subkutan İmmünglobülin Alan Hastalarımızın Klinik İzlemi

Primer immün yetmezliklerde enfeksiyonların önlenmesinde immünglobülin tedavisi uygulanmaktadır. Son yıllarda intravenöz yerine subkutan tedavi tercih edilmektedir. Bu çalışmada merkezimizde primer immün yetmezlik nedeniyle subkutan immünglobülin tedavisine geçilen hastaların demografik ve klinik özellikleri verilmiştir. 2014-2017 yılları arasında Eskişehir Osmangazi Üniversitesi Tıp Fakültesi Çocuk Alerji ve İmmünoloji kliniğinde primer immün yetmezlik tanısı ile subkutan tedaviye geçilen hastaların dosyaları retrospektif olarak incelendi. Çalışmaya primer immün yetmezlik tanılı 15 hasta alındı. Hastaların 9 (%60)’u erkek idi, yaşları en düşük 10 ay - en yüksek 15 yıl olarak saptandı. Hastaların üç tanesinde ataksi telenjektazi (%20), üç tanesinde ortak değişken immün yetmezlik (%20), 3 tanesinde X’e bağlı agamaglobülinemi (%20), bir tanesinde otozomal resesif agamaglobülinemi (%6.6), iki tanesinde sınıflandırılamayan hipogamaglobülinemi (%13.2), bir tanesinde geçici hipogamaglobülinemi (%6.6), bir tanesinde hiperimmünglobülin E sendromu (%6.6) ve bir tanesinde hiperimmünglobülin M sendromu (%6.6) tanısı mevcuttu. Ataksi telenjektazi ile takip edilen 3 hastanın 2’si subkutan immünglobülin tedavisinin 1. yılında geçirdikleri enfeksiyon nedeniyle hastaneye yatırılarak tedavi edildiler. Diğer hastaların 2 yıllık izlem sürecinde hastane yatışı gerektirecek enfeksiyonu kaydedilmedi. Tüm hastalar sonraki takiplerinde subkutan yolu tercih edeceklerini belirttiler. Sonuç olarak; subkutan yolla immünglobülin tedavisi, etkin, kolay ve emniyetli bir seçenektir.

Correlations Among Subcutaneous Immunoglobulin Dosage, ImmunoglobulinG Serum Pre-infusional Levels and Body Mass Index in Primary Antibody Deficiency Patients: A Pooled Analysis from the SHIFT/IBIS Studies.

Background and ObjectiveIn recent years, two Italian non-interventional studies evaluated subcutaneous immunoglobulin (SCIG) treatment in patients affected by primary antibody deficiency (PAD). The SHIFT study considered patients who were treated with intravenous immunoglobulin (IVIG) or SCIG 16% (Vivaglobin®) and then replaced this therapy with weekly treatments of SCIG 20% (Hizentra®). The IBIS study evaluated patients previously taking a weekly SCIG 20% regimen, who instead began therapy with biweekly SCIG 20% to assess the correlation between the dose of immunoglobulin G (IgG) administered and the body mass index (BMI) of patients, determine if there is a need for dosage adjustments on a BMI basis, and identify the predictors of serum IgG trough levels in our cohort.MethodsIn this study, we analyzed the pooled data of 109 PAD patients enrolled in the SHIFT and IBIS studies. Only prospective phases were considered.ResultsThe total monthly SCIG dose showed comparable trends among weight categories, except for underweight patients. When we considered the monthly SCIG dosage per kilogram of body weight, a significant decreasing trend according to BMI was observed. Data on IgG trough levels were available for 88 patients, with a mean IgG serum level of 8.4 ± 1.6 g/L. A stepwise regression model revealed that the mean monthly dosage of SCIG 20% (p = 0.04248) and the mean monthly dosage of IgG per kilogram of body weight were the only two independent predictors associated with IgG trough levels. No association was found between BMI and IgG trough levels.ConclusionsThese findings support the concept that the cumulative monthly dose of SCIG and the dose of SCIG per kilogram of body weight affect IgG trough levels in PAD patients, irrespective of BMI.