Subcutaneous Immunoglobulin Replacement Therapy with Hizentra® is Safe and Effective in Children Less Than 5 Years of Age.

BACKGROUND:Hizentra® (IGSC 20%) is a 20% liquid IgG product approved for subcutaneous administration in adults and children 2 years of age and older who have primary immunodeficiency disease (PIDD). There is limited information about the use of IGSC 20 % in very young children including those less than 5 years of age. METHODS:A retrospective chart review involved 88 PIDD infants and children less than 5 years of age who received Hizentra®. RESULTS:The mean age at the start of Hizentra® was 34 months (range 2 to 59 months). IGSC 20 % was administered weekly to 86 infants (two additional infants received twice weekly and three times weekly infusions, respectively) and included an average of 63 infusions (range 6-182) for an observation period up to 45.5 months. Infusion by manual delivery occurred in 15 patients. The mean dose was 674 mg/kg/4 weeks. The mean IgG level was 942 mg/dL while on IGSC 20 %, compared to a mean trough IgG level of 794 mg/dL (p < 0.0001) during intravenous or subcutaneous IgG administration prior to IGSC 20 %. Average infusion time was 47 (range 5-120) minutes, and the median number of infusion sites was 2 (range 1-4). Local reactions were mostly mild and observed in 36/88 (41%) children. No serious adverse events were reported. A significant increase in weight percentile (7 % ± 19.2, p = 0.0012) among subjects was observed during IGSC 20% administration. The rate of serious bacterial infections was 0.067 per patient-year while receiving IGSC 20%, similar to previously reported efficacy studies. CONCLUSIONS:Hizentra® is effective in preventing infections, and is well tolerated in children less than age 5 years

Home-Based Family Intervention for Low-Income Children With Asthma: A Randomized Controlled Pilot Study

Low-income African American children have disproportionately higher asthma morbidity and mortality. Education alone may not address barriers to asthma management due to psychosocial stress. This study evaluated the efficacy of a home-based family intervention integrating asthma education and strategies to address stress using a community-based participatory research model. Children age 8 to 13 with poorly controlled asthma and their caregivers were recruited from an urban hospital and an asthma camp. Caregivers with elevated scores on a stress measure were enrolled. Forty-three families were randomized to the 4- to 6-session Home Based Family Intervention (HBFI) or the single session of Enhanced Treatment as Usual (ETAU). All families received an asthma action plan and dust mite covers; children performed spirometry and demonstrated MDI/spacer technique at each home visit. The HBFI addressed family-selected goals targeting asthma management and stressors. Asthma management, morbidity, family functioning, and caregiver stress were assessed at baseline, postintervention, and 6 months after the intervention. ED visits and hospitalizations were ascertained by medical record review for a year after intervention completion. Only one child (5%) in HBFI had an asthma-related hospitalization compared to 7 patients (35%) in ETAU in the year following intervention. Participants in both groups demonstrated improved asthma management and family functioning, and reduced ED visits, symptom days, missed school days, and caregiver stress, but there were no differential treatment effects. The results suggest that a home-based intervention addressing medical and psychosocial needs may prevent hospitalizations for children with poorly controlled asthma and caregivers under stress

Recombinant human hyaluronidase facilitated subcutaneous immunoglobulin treatment in pediatric patients with primary immunodeficiencies: long-term efficacy, safety and tolerability.

AimTo 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 & methodsPatients with primary immunodeficiency diseases were included in the studies. IGHy was administered every 3 or 4 weeks.ResultsValidated acute serious bacterial infections were reported at 0.08/patient-year (four pneumonia episodes in three patients). No serious adverse drug reaction (ADR) was reported, and rates of local and systemic ADRs were low (0.09/infusion and 0.1/infusion). Infection rates were low (3.02/patient-year) with sustained Ig trough levels (median: 1009 mg/dl). Of 674 IGHy infusions, 97.2% required no change of administration due to ADR, in most (82.5%) with one infusion site. No patient developed neutralizing anti-rHuPH20 antibodies. Postpivotal study, 100% of patients aged <14 years or their caregivers and 85.7% of patients aged 14 to <18 years expressed preference for IGHy compared with Ig administered intravenously or Ig administered subcutaneously.ConclusionThese studies, with the longest (maximum: 3.3 years) duration of any reported Ig replacement trials in children with primary immunodeficiency diseases, showed low infection, local and systemic reaction rates along with well-tolerated infusions given in a single site