Trough tobramycin concentrations were significantly influenced in some CF patients (45%), suggesting that timing of the inhaled dose should be considered when interpreting PK measures of IV tobramycin dosing.

Teriparatide is a human parathyroid hormone analog approved for the treatment of osteoporosis in adult patients. Its use for hypocalcemia and hypoparathyroidism in the pediatric population is described through case reports and small case series; however, larger studies that demonstrate long-term efficacy and safety are limited. At our institution, a 4-month-old premature (gestational age: 32 weeks) infant with multiple congenital anomalies, functional athymia, and severe hypoparathyroidism and receiving calcitriol, vitamin D, and calcium carbonate supplementation was initiated on subcutaneous injection of teriparatide. During the course of treatment, her calcium carbonate, vitamin D, and calcitriol supplementation requirements substantially decreased. Teriparatide effectively increased serum ionized calcium concentrations and decreased serum phosphorus concentrations in the present case-study over a 6-month period. Teriparatide was well tolerated, and no evidence of hypercalcemia was observed throughout treatment.

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Background: The goal of allogeneic hematopoietic stem cell transplantation (HSCT) for bone marrow failure syndromes is stable and adequate engraftment of donor cells, low treatment-related mortality, and absent or minimal GVHD. Methods: We retrospectively analyzed outcomes for 10 pediatric patients (ages 10 months to 19 years, median 5.5 years) with bone marrow failure syndromes-7 with severe aplastic anemia (SAA) and 3 with congenital amegakaryocytic thrombocytopenia (CAMT)-who received a reduced intensity conditioning (RIC) regimen with fludarabine, thiotepa, melphalan, and rabbit ATG (rATG) prior to HSCT between 2009 and 2017. The RIC regimen consisted of fludarabine 30 mg/ m 2 /day × 5 days on day −9 to −5, thiotepa 5 mg/kg/dose every 12 hours on day −4, rabbit ATG with a 1 mg/kg test dose on day −4 followed by 3 mg/kg/day on days −3 to −1, and melphalan 70 mg/m 2 daily × 2 days on days −3 and −2. Tacrolimus (from day −2 onward) and mycophenolate (from day 0 onward) were used for acute GVHD (aGVHD) prophylaxis. Results: Seven patients received stem cells from a 10/10 matched sibling donor (MSD). Five underwent MSD bone marrow transplantation (BMT); 2 had MSD peripheral blood stem cell transplantation (PBSCT). Three patients received a 10/10 matched unrelated donor (MUD) BMT. No patients experienced veno-occlusive disease (VOD), delayed engraftment, or graft failure. All patients were transfusion independent by day 100. The median time to neutrophil engraftment was 16.2 days (range 10-26 days). Five patients had no aGVHD; 2 had grade I and 3 had grade II aGVHD. No patients experienced grade III or IV aGVHD. Four patients had no chronic GVHD (cGVHD), while 2 experienced limited and 4 had extensive cGVHD. Seven of 10 patients had discontinued systemic immunosuppressive therapy (IST) after a median of 308 days post-HSCT (S.D. ±236). Two patients, both <1-year post-HSCT, were undergoing scheduled weaning of IST. One patient, a recipient of a MSD PBSCT with extensive cGVHD, was still on IST 659 days post-HSCT. With a median follow-up of 650 days, the probability of overall survival at both 100 days and 1 year was 100%. Conclusion: Our data suggest that RIC with fludarabine, thiotepa, melphalan, and rATG prior to MSD and MUD HSCT for SAA and CAMT is effective with a tolerable safety profile. Better IST is needed to decrease the incidence and severity of cGVHD in this population.