Context Daily growth hormone (GH) injections can be burdensome for patients and carers. Somapacitan is a long-acting, reversible albumin-binding GH derivative in development for once-weekly administration in patients with growth hormone deficiency (GHD). Objective The objective of this study is to evaluate the efficacy, safety, and tolerability of once-weekly somapacitan vs once-daily GH. Design REAL 3 is a multicenter, randomized, controlled, double-blind (somapacitan doses), phase 2 study with a 26-week main and 26-week extension phase (NCT02616562). Setting This study took place at 29 sites in 11 countries. Patients Fifty-nine GH treatment-naive prepubertal children with GHD were randomly assigned; 58 completed the trial. Interventions Interventions comprised 3 somapacitan doses (0.04 [n = 16], 0.08 [n = 15], or 0.16 mg/kg/wk [n = 14]) and daily GH (0.034 mg/kg/d [n = 14]), administered subcutaneously. Main Outcome Measures The primary end point was height velocity (HV) at week 26. Secondary efficacy end points included HV SD score (SDS) and insulin-like growth factor-I (IGF-I) SDS. Results At week 26, mean (SD) annualized HV for the somapacitan groups was 8.0 (2.0), 10.9 (1.9), and 12.9 (3.5) cm/year, respectively, vs 11.4 (3.3) cm/year for daily GH; estimated treatment difference (somapacitan 0.16 mg/kg/week—daily GH): 1.7 [95% CI –0.2 to 3.6] cm/year. HV was sustained at week 52, and significantly greater with somapacitan 0.16 mg/kg/week vs daily GH. Mean (SD) change from baseline in HV SDS at week 52 was 4.72 (2.79), 6.14 (3.36), and 8.60 (3.15) for the somapacitan groups, respectively, vs 7.41 (4.08) for daily GH. Model-derived mean (SD) IGF-I SDS for the somapacitan groups was −1.62 (0.86), −1.09 (0.78), and 0.31 (1.06), respectively, vs −0.40 (1.50) observed for daily GH. Safety and tolerability were consistent with the profile of daily GH. Conclusions In children with GHD, once-weekly somapacitan 0.16 mg/kg/week provided the closest efficacy match with similar safety and tolerability to daily GH after 26 and 52 weeks of treatment. A short visual summary of our work is available (1).
A case of granular cell tumor of the urinary bladder is reported. A 48-year-old Japanese man with a bladder tumor underwent transurethral resection of the tumor and microscopic examination showed a granular cell tumor. Immunohistochemical staining for neuron specific enolase and S100 protein was positive, and it was suggested that the granular cell tumor was derived from Schwann cells.
Our study suggests that immunological responses are involved in the development of interstitial cystitis symptoms. IPD-1151T could be a new oral agent for treatment of voiding symptoms and bladder pain in patients with interstitial cystitis.
Pyridinoline cross-linked carboxyterminal telopeptide might assist PSA and bone scintigraphy in monitoring metastatic bone activity of prostatic cancer.
Summary To clarify the meaning and clinical value of bone formation markers in bone metastasis from prostate cancer, we investigated the bone formation markers carboxy-terminal propeptide of type procollagen (PICP), bone-specific alkaline phosphatase (BAl-p) and osteocalcin, so-called bone gla protein (BGP) in 43 prostate cancer patients with and 46 patients without overt bone metastasis. Patients with bone metastasis were evaluated repeatedly by bone scan at intervals of 3-6 months. The expression patterns of bone formation markers in patients with progression of bone metastasis became dissociated; BAl -p and PICP were elevated in patients with progression of bone metastasis but BGP was not. Instead, BGP showed slight elevation in patients with improvement and complete remission of bone metastasis. PICP, BAl-p and BGP are all bone formation markers, but each marker appears in a different phase of bone formation: PICP appears in proliferation phase, BAl -p appears in matrix maturation phase and BGP appears in late bone formation phase. Our findings that BGP was not elevated in progression of bone metastasis and that it increased slightly with improvement and complete remission of bone metastasis may imply that the bone formation that occurs in blastic bone metastasis is different from normal bone formation.Keywords: prostate cancer; bone metastasis; carboxy-terminal propeptide of type procollagen; bone-specific alkaline phosphatase; bone gla protein Bone metastasis from prostate cancer is frequently osteoblastic in nature, and it often shows up as osteoplastic changes on plain radiography. Recently, various bone metabolic markers of both formation and resorption have been identified. Bone resorption markers have been proven to be useful in the bone metastasis of prostate cancer (Kylmala et al, 1995; Maeda et al, 1996). However, the meaning of bone formation markers is not fully understood in bone metastasis of prostate cancer. Various bone formation markers have been found to be associated with certain phases of bone formation. The carboxy-terminal propeptide of type I procollagen (PICP) is believed to be a marker of early bone formation and it generally appears during osteoblast proliferation. Bone-specific alkaline phosphatase (BAl-p) is a marker of the middle stage of bone formation and it appears in the matrix maturation phase. Osteocalcin, so-called bone gla protein (BGP), is a marker of late bone formation and it appears in the mineralization phase (Stein et al, 1990;Risteli and Risteli, 1993;Zhou et al, 1994;Calvo et al, 1996). To clarify the meaning of bone formation markers, we investigated these three markers in prostate cancer patients with and without overt bone metastasis. PATIENTS AND METHODSBetween October 1994 and April 1996, 43 prostate cancer patients with and 46 patients without overt bone metastasis were studied with respect to bone formation markers. Of the 46 patients without Institute Hospital, 1-37-1 Kami-lkebukuro, Toshima-ku, Tokyo 170, Japan overt bone metastasis, 29 had a history of r...
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