Treatment and Prognosis of Inflammatory Pseudotumor of the Skull Base.

Background Inflammatory pseudotumor (IPT) of the skull base is a rare, locally destructive lesion managed with a variety of treatments. We explore the impact of treatment on outcome and assess the prognosis of IPT. Methods This is a retrospective review of IPT of the skull base at a tertiary academic medical center. The primary outcome was radiographic progression after treatment. Outcome versus tumor location was also examined and a prognostic model was developed using a logistic regression. Results The demographics of 21 patients with IPT are reported. Treatment consisted of corticosteroids (in 80.1% of patients), disease modifying antirheumatic drugs (DMARDs; 33.3%), surgical resection (28.6%), radiation (23.8%), antibiotics (14.3%), chemotherapy (rituximab; 9.5%), and antivirals (4.8%). At 50.7 months, 50.8% had radiographic progression. Local therapy trended toward having a better response than systemic therapy ( p = 0.60). IPT of the orbit required 2.4 treatment modalities, compared with 2.0 for pharyngeal IPT, and 1.3 for posterior skull base masses ( p = 0.14). A total of 75% orbital IPT underwent radiographic progression, compared with 71% of pharyngeal IPT and 50% of posterior skull base masses ( p = 0.62). Sixteen patients were used to create the logistic model of radiographic progression. The Cox-Snell R 2 was 0.71 ( p = 0.03). No individual variables were statistically significant. Conclusion To our knowledge, this is among the largest sample of cases describing the presentation, treatment, and prognosis of IPT of the skull base. Our data suggest that there may be an improved response with local therapy over systemic therapy and better prognosis among posterolateral skull base masses.

Desmoid tumors of the head and neck in the pediatric population: Has anything changed?

INTRODUCTION: Pediatric head and neck desmoid tumors are rare neoplasms that can cause significant morbidity due to infiltration of vital anatomic structures. The goal of this study is to review presentation, evaluation, and management of these tumors. METHODS: Retrospective study of children with head and neck desmoid tumors treated from 1999 to 2018 and literature review. RESULTS: 11 patients (5 boys, 6 girls) were included. Presentation included firm neck mass (n = 8), trismus (n = 2) and tongue lesion (n = 1). All patients had preoperative imaging with CT (n = 2), MRI (n = 1) or both (n = 8). Five patients underwent needle biopsy, five had open biopsy and one was diagnosed on pathology from primary excision. Seven patients were treated by primary surgical resection, with positive surgical margins in six cases due to proximity to vital neurovascular structures. None needed chemotherapy, had disease recurrence or progression. Three patients with unresectable disease were treated with chemotherapy. One patient was monitored with imaging without any treatment and did not have disease progression. Follow-up ranged from 6 months to 6 years (median 21 months). Ten patients (7 surgical, 2 chemotherapy, 1 observation) were either disease-free or had stable disease at last follow-up. CONCLUSION: Pediatric head and neck desmoid tumors, though rare and histologically benign, are locally infiltrative and aggressive. When feasible, surgical treatment results in good disease control despite positive margins. A balance between achieving negative margins and minimizing functional deficits should be considered. Chemotherapy can be successfully utilized in patients where surgery entails a high risk of morbidity and mortality.

Familial and genetic factors in laryngeal cleft: Have we learned anything?

INTRODUCTION: Laryngeal clefts are rare congenital anomalies characterized by failed fusion of the posterior cricoid lamina or incomplete development of the tracheoesophageal septum. While most cases are sporadic, laryngeal cleft may be associated with other congenital anomalies or syndromes. Though not frequently reported, familial occurrence of laryngeal cleft has been noted in our clinical experience. The goal of this research is to describe the existing literature and our own experience surrounding familial occurrence of laryngeal cleft that may help elucidate its underlying genetic basis. METHODS: Comprehensive literature search was conducted and retrospective chart review was performed on 8 sets of siblings diagnosed at our institution. Data assessed included demographics, type of cleft, and genetic findings. RESULTS: Laryngeal cleft appears to be mostly sporadic. We evaluated data at our institution over a 10-year period and identified 19 patients from 8 families demonstrating familial occurrence of laryngeal cleft. Six (75%) families had two affected siblings, one family (12.5%) had three affected siblings, and one family (12.5%) had four affected siblings. There was no evidence of sex predilection, with half the patients being male (10/19, 52.6%). Fourteen patients (73.7%) had Type 1 clefts and five (26.3%) had Type 2 clefts. Genetic findings were available for review in five patients from three families. CONCLUSION: Beyond a few known syndromes, laryngeal cleft has largely been thought to be sporadic. However, findings from the existing literature and our own experience with familial laryngeal cleft in eight families suggest additional genetic factors are yet to be elucidated.

Live-animal tracking of individual haematopoietic stem/progenitor cells in their niche.

Stem cells reside in a specialized, regulatory environment termed the niche that dictates how they generate, maintain and repair tissues. We have previously documented that transplanted haematopoietic stem and progenitor cell populations localize to subdomains of bone-marrow microvessels where the chemokine CXCL12 is particularly abundant. Using a combination of high-resolution confocal microscopy and two-photon video imaging of individual haematopoietic cells in the calvarium bone marrow of living mice over time, we examine the relationship of haematopoietic stem and progenitor cells to blood vessels, osteoblasts and endosteal surface as they home and engraft in irradiated and c-Kit-receptor-deficient recipient mice. Osteoblasts were enmeshed in microvessels and relative positioning of stem/progenitor cells within this complex tissue was nonrandom and dynamic. Both cell autonomous and non-autonomous factors influenced primitive cell localization. Different haematopoietic cell subsets localized to distinct locations according to the stage of differentiation. When physiological challenges drove either engraftment or expansion, bone-marrow stem/progenitor cells assumed positions in close proximity to bone and osteoblasts. Our analysis permits observing in real time, at a single cell level, processes that previously have been studied only by their long-term outcome at the organismal level.