I. Melamed

Purpose To achieve reductions in infusion time, infusion sites, and frequency, a prospective, open-label, multicenter, Phase 3 study evaluated the safety, efficacy, and tolerability of subcutaneous immunoglobulin (SCIG) 16.5% (Cutaquig®, Octapharma) at enhanced infusion regimens. Methods Three separate cohorts received SCIG 16.5% evaluating volume...

Purpose To achieve reductions in infusion time, infusion sites, and frequency, a prospective, open-label, multicenter, Phase 3 study evaluated the safety, efficacy, and tolerability of subcutaneous immunoglobulin (SCIG) 16.5% (Cutaquig®, Octapharma) at enhanced infusion regimens. Methods Three separate cohorts received SCIG 16.5% evaluating volume...

[This corrects the article DOI: 10.3389/fimmu.2019.00040.].

A prospective study and its long-term extension examined whether weekly treatment of patients with primary immunodeficiencies (PIDs) with a 16.5% SCIg (cutaquig®) confers acceptable efficacy, safety, and tolerability over a follow-up of up to 238 weeks (>4 years). Seventy-five patients received 4,462 infusions during up to 70 weeks of follow-up in...

Background: Subcutaneous human immunoglobulin (16.5%; octanorm/cutaquig ® ) was efficacious and well tolerated in patients with primary immunodeficiencies in a Phase III study. A subanalysis of pediatric data is presented here. Materials & methods: Children (2–16 years) previously treated with intravenous human immunoglobulin received weekly subcut...

Objectives: Pediatric acute-onset neuropsychiatric syndrome (PANS) is a clinical diagnosis in children who have an acute manifestation of varied neuropsychiatric symptoms, including obsessive compulsive disorder, eating disorders, tics, anxiety, irritability, and problems with attention/concentration. PANS may develop as a result of a postinfectiou...

Introduction: Subcutaneously administered immunoglobulin (SCIG) is increasingly used to treat patients with primary immunodeficiencies (PIDs). Octanorm (marketed as cutaquig® in USA and Canada) is a new 16.5% solution of human SCIG, manufactured by a process based on that of the intravenous preparation (IVIG) octagam®. Objectives: To investigate th...

Aim: To assess the safety and efficacy of an intravenous immunoglobulin (IVIG) 10% preparation (Panzyga®; Octapharma AG, Lachen, Switzerland) in predominantly antibody-deficient children with primary immunodeficiency disease. Methods: Data from two prospective, open-label and noncontrolled multicenter Phase III studies of IVIG 10% that included...

Intravenous immunoglobulin (IVIG) therapy is commonly used to treat patients with primary antibody deficiency. This prospective, open-label, non-randomised, multicentre, phase III trial investigated the pharmacokinetics of a new 10% liquid IVIG product (panzyga®; Octapharma) in 51 patients aged 2-75 years with common variable immunodeficiency (n =...

Lay summary: Since research has demonstrated a link between autism spectrum disorder (ASD) and immune dysfunction, this study investigated the efficacy and tolerability of intravenous immunoglobulin (IVIG) infusion in children with ASD. Fourteen patients received IVIG treatment and were assessed using standardized cognitive and behavioral tests. F...

Background: Approximately two-thirds of patients with chronic inflammatory demyelinating polyneuropathy (CIDP) need long-term intravenous immunoglobulin. Subcutaneous immunoglobulin (SCIg) is an alternative option for immunoglobulin delivery, but has not previously been investigated in a large trial of CIDP. The PATH study compared relapse rates i...

Purpose: To assess the efficacy and safety of panzyga® (intravenous immunoglobulin 10%) in preventing serious bacterial infections (SBIs) in patients with primary immunodeficiency diseases (PIDs), a prospective, open-label, multicenter, phase 3 study and an open-label extension study were undertaken. Methods: Initially, the study drug (infusion...

p> Background: Cysteinyl leukotriene levels are elevated in patients with atopic dermatitis, which can lead to eosinophilic infiltration of the gastrointestinal tract. Objective: We examined the role that montelukast (a leukotriene receptor antagonist) might play in improving symptoms of atopic dermatitis induced by food allergies. Methods: We c...

PurposeThis phase 3, multicenter, open-label, randomized, two-period, crossover bioequivalence trial evaluated the safety, tolerability, and pharmacokinetics of intravenous immunoglobulins (IVIGs) Gammaplex 5% and Gammaplex 10% in 33 adults and 15 children with primary immunodeficiency diseases (PIDs). Methods Eligible adults received five Gammaple...

Aim: To assess the long-term efficacy, safety and tolerability of recombinant human hyaluronidase-facilitated subcutaneous infusion of immunoglobulin (Ig) (fSCIG; HYQVIA(®); IGHy) in children aged <18 years. Patients & methods: Patients with primary immunodeficiency diseases were included in the studies. IGHy was administered every 3 or 4 weeks....

Patients with primary immunodeficiency disease (PIDD) typically require life-long intravenous (IV) or subcutaneous (SC) immunoglobulin (Ig) replacement therapy to prevent recurrent infections. The efficacy, safety, and pharmacokinetics of a highly concentrated (20 %) Ig preparation for SC administration (IGSC 20 %) were evaluated in a prospective t...

Purpose: Treatment of primary immunodeficiency diseases (PIDD) with subcutaneous (SC) infusions of IgG preceded by injection of recombinant human hyaluronidase (rHuPH20) (IGHy) to increase SC tissue permeability was evaluated in two consecutive, prospective, non-controlled, multi-center studies. Methods: Subjects >4 years of age received SC IgG...

Purpose: Immune globulins for IgG supplementation have been produced for over 35 years with essentially no differentiating features regarding their specific antibody composition. Furthermore, the compositions of plasma donor pools used for IG manufacturing are not standardized. While all immune globulin products meet the specifications set by the...

Purpose: This phase 4, multicenter, open-labeled, single-sequence, crossover study in pediatric patients (ages 2 to 16) with primary immunodeficiency disease (PID) evaluated the pharmacokinetics, safety, and tolerability for subcutaneously (SC) administered 10 % caprylate/chromatography purified human immune globulin injection (IGIV-C, GAMUNEX®) c...

Background: fSCIG can be administered at similar doses/volumes and dosing intervals as intravenous immunoglobulin G (IgG) (IVIG) but, similar to conventional subcutaneous IgG, is associated with a lower risk of systemic and higher risk of local adverse reactions (ARs). Objective: We report local AR rates over time in patients with PID aged ≥ 16 ye...

The interaction between infectious pathogens and the immune system has been a focus of research for many years. However, the failure of re-recognition or immune memory of infectious pathogen remains a clear mystery A memory B cell defect coupled with low levels of C1-INH and/or C1-INH function-failure of both the innate and adaptive immune componen...

The association between ADHD and allergy remains controversial. Our previous findings suggest that nerve growth factor may link the nervous and immune systems. The primary objective of this study was to determine if a combination of cetirizine + methylphenidate is effective in children with comorbid ADHD and allergic rhinitis. We also examined the...

This open-label multicentre study evaluated Gammaplex® 5%, a human intravenous immunoglobulin (IVIG) 5% liquid, in 25 children and adolescent patients (aged 3–16 years) with primary immunodeficiency diseases (PIDs). Subjects received Gammplex 5% (at doses of 300–800 mg/kg/infusion) for 12 months, with a 3-month follow up. The primary efficacy endpo...

Objective: To evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB033 (anti-LINGO-1 monoclonal antibody) in healthy volunteers and participants with multiple sclerosis (MS). Methods: In 2 separate randomized, placebo-controlled studies, single ascending doses (SAD; 0.1-100 mg/kg) of BIIB033 or placebo were administered via IV inf...

Objective: Tolerability of IVIG is very good. However, a subpopulation of patients have difficulty tolerating a high concentration product, such as a 10% IVIG formulation, due to systemic adverse events (AEs), such as headaches, fatigue, fever and muscle aches. While many patients experience fewer systemic AEs with SCIG, it requires weekly infusion...

Background: Three small trials suggest that intravenous immunoglobulin can affect biomarkers and symptoms of mild-to-moderate Alzheimer's disease. We tested the safety, effective dose, and infusion interval of intravenous immunoglobulin in such patients. Methods: We did a multicentre, placebo-controlled phase 2 trial at seven sites in the USA an...

Research on the role of subcutaneous immunoglobulin in primary immunodeficiency disease (PIDD) is ongoing. We analyzed pivotal studies for four subcutaneous immunoglobulin products: IGSC 10% (Gammagard® Liquid), IGIV-C 10% (Gamunex®-C), IGSC 16% (Vivaglobin®) and IGSC 20% (Hizentra®). To identify similarities and differences between products, we ex...

Subcutaneous immunoglobulin (IGSC) replacement therapy for primary immunodeficiency (PI) is equally efficacious to intravenous immunoglobulin (IGIV), induces fewer systemic reactions, and may be self-infused. Limited SC infusion volumes and reduced bioavailability, however, necessitate multiple infusion sites, more frequent treatment, and dose adju...

An investigational 10% liquid intravenous immunoglobulin (IVIG) was studied in 63 patients with primary immunodeficiency (PID) at 15 study sites. Patients were treated every 3 or 4 weeks with 254-1029 mg/kg/infusion of IVIG. Overall, Biotest-IVIG infusions were well tolerated. The proportion of infusions that were associated with adverse events dur...

Introduction: A disadvantage of subcutaneous (SC) treatment is decreased bioavailability versus intravenous (IV) immunoglobulin (IGIV). Human immunoglobulin with rHuPH20 (IGHy) improves bioavailability versus SC formulations of immunoglobulin G (IGSC), minimizes the need for multiple infusion sites, and enables infusion at rates and frequencies equ...

Background and objectives: Immunoglobulin replacement is a standard therapy for patients with primary immunodeficiencies. Subcutaneous administration of immunoglobulin offers more constant IgG levels than intravenous administration and simplifies administration for some patients. Use of L-proline as an excipient contributes to the stability of hig...

A multi-center, prospective, open-label study was conducted in primary immunodeficiency disease patients to determine the tolerability and pharmacokinetics of a 10% liquid IgG preparation administered subcutaneously. Forty-nine subjects (3-77 years old) were enrolled. Pharmacokinetic equivalence of subcutaneous treatment was achieved at a median do...

Subcutaneous human IgG (SCIG) therapy in primary immunodeficiency (PID) offers sustained IgG levels throughout the dosing cycle and fewer adverse events (AEs) compared to intravenous immunoglobulin (IVIG). A phase I study showed good local tolerability of IgPro20, a new 20% liquid SCIG stabilized with L-proline. A prospective, open-label, multicent...

Intravenous immunoglobulin (IVIg) is used in treating immunodeficiencies and autoimmune or inflammatory disorders. As manufacturing processes and storage can alter IgG molecules, pharmacokinetic assessments are important for new preparations. Thus, we studied pharmacokinetics of IgPro10, a new 10% liquid IVIg product stabilised with l-proline, in p...

Omenn's syndrome is a fatal, autosomal-recessive combined immune deficiency characterized by several erythematous exfoliative eruptions, lymphadenopathy, hepatosplenomegaly, and elevated eosinophil count. In some of these patients an expansion of CD3+CD4−CD8− double negative (DN) T cell population was observed. We show here that the DN population r...

Subjects with primary immune deficiency diseases treated with intravenous immunoglobulin (n=42) received intravenous infusions of Carimune NF Liquid every 3-4 weeks for 6 months without routine premedication. The mean dose/patient/infusion was 278.5-800.7 mg/kg. Also, 80.4% of infusions achieved maximum rates of >or=3.5 mg/kg/min; 32% of infusions...

Gaucher's disease (GD) is an inborn error, autosomal recessive lysosomal lipid storage disorder characterized by the lack of the enzyme glucocerebrosidase. We observed some abnormalities in the plasma of a traumatized patient with GD. We report of a traumatized patient with GD that developed a severe systemic immune response during the course of an...

The present clinical study was designed to evaluate the efficacy, pharmacokinetics and safety of a new 10% liquid intravenous immune globulin in patients with primary immunodeficiency diseases. Sixty-one adults and children with primary immuno-deficiency diseases received doses of 300-600 mg/kg body weight every 21-28 days for 12 months. No validat...

The etiology of multiple sclerosis (MS) remains unclear. To determine if autoantibodies to myelin basic protein (MBP) are produced during parvovirus B19 infection, a competitive ELISA was performed using plasma from MS patients exhibiting high IgG titers for parvovirus. Our results showed the addition of MBP decreased the binding of IgG to B19 anti...