ABSTRACT
BACKGROUND: The onset of bronchiolitis obliterans (BO) as a pulmonary manifestation of chronic graft vs host disease dramatically changes the prognosis of children undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). This study aimed to evaluate the overall survival (OS) of children with BO treated with imatinib mesylate (IM). METHODS: This study included children who underwent allo-HSCTs between January 2000 and December 2016. RESULTS: Among 345 patients who underwent HSCTs, 293 were evaluable for BO and 26 (8.9%) developed BO. The cumulative incidence of BO was 4.8% (95% confidence interval [CI], 2.8-7.5) at 1 year and 7.7% (95% CI, 5.1-11.1) at 3 years after transplantation. In the group of HSCTs (n = 67) complicated by chronic GvHD (c-GVHD), the incidence rate of BO was 38.8%. In total, 96.1% of patients with BO had c-GvHD worse than moderate grade, which was present in 70.7% of patients without BO (P = .011). The mortality rates were 46.1% in the BO group and 27.4% in the group without BO. Half of the patients with BO (n = 13) received IM, and the overall response rate was 76.9%. Four years after HSCT, OS was 42.6% (95% CI, 18.2-65.3) in the group without IM and 83.3% (95% CI, 27.3-97.5) in the group with IM. CONCLUSIONS: BO after HSCT in the pediatric population has a high incidence and mortality rate. In terms of overall response and tolerability, this study showed relevant improvements in the prognosis of children with BO after the introduction of IM. Further prospective studies among children are needed to confirm these results.
Subject(s)
Bronchiolitis Obliterans/drug therapy , Graft vs Host Disease/drug therapy , Hematopoietic Stem Cell Transplantation/adverse effects , Imatinib Mesylate/therapeutic use , Adolescent , Bronchiolitis Obliterans/etiology , Child , Child, Preschool , Female , Graft vs Host Disease/complications , Humans , Incidence , Male , Prognosis , Transplantation, HomologousABSTRACT
OBJECTIVE: To investigate the frequency of ultrasound (US)-detectable involvement of the subtalar joint (STJ), to compare clinical versus US assessment of the STJ, and to compare different scanning approaches to the STJ in juvenile idiopathic arthritis (JIA). METHODS: Clinical and US assessments were performed independently in 50 ankles with clinically active JIA. US abnormalities of the STJ were investigated using a lateral, medial, and posterior scanning approach and scored semiquantitatively. Agreement was tested using kappa statistics. A control group of 10 healthy subjects was examined. RESULTS: Clinical and US evaluations detected synovitis in 24 of 50 (48.0%) and 27 of 50 (54.0%) of STJs, respectively. US detected synovitis in 10 of 26 STJs (38.5%) recorded as normal on clinical evaluation, but was negative in 7 of 24 STJs (29.2%) diagnosed as having involvement on clinical examination. Agreement between clinical and US assessments was fair (κ = 0.32). US abnormalities were more frequently detectable using the lateral scanning approach. All patients with US abnormalities in the medial and/or posterior side of the STJ had also US abnormalities on the lateral scanning approach, but the reverse was not true. Intra- and interobserver agreements for the lateral scanning approach were satisfactory for both detecting involvement and scoring US abnormalities. None of the 17 STJs of healthy controls showed US abnormalities. CONCLUSION: US may increase the precision of the evaluation of the STJ in JIA. The observed high frequency of STJ involvement on US suggests to include this joint in US scanning protocols devised for children with JIA. Synovitis is more frequently detected using the lateral scanning approach.