Richard G. Azizkhan

Sirolimus for the treatment of complicated vascular anomalies in children.

Vascular anomalies comprise a diverse group of diagnoses. While infantile hemangiomas are common, the majority of these conditions are quite rare and have not been widely studied. Some of these lesions, though benign, can impair vital structures, be deforming, or even become life‐threatening. Vascular tumors such as kaposiform hemangioendotheliomas (KHE) and complicated vascular malformations have proven particularly difficult to treat.

Differentiated thyroid cancer : Determinants of disease progression in patients <21 years of age at diagnosis : A report from the surgical discipline committee of the children's cancer group

OBJECTIVE This study was done to define the extent of disease and evaluate the effect of staging and treatment variables on progression-free survival in patients with differentiated thyroid carcinoma who were less than 21 years of age at diagnosis. SUMMARY BACKGROUND DATA Differentiated thyroid cancer in young patients is associated with early regional lymph node involvement and distant parenchymal metastases. Despite this, the overall long-term survival rate is greater than 90%, which suggests that biologic rather than treatment factors have a greater effect on outcome. METHODS Variables analyzed for their impact on progression-free survival in a multi-institutional cohort of 329 patients included age, antecedent thyroid irradiation, extrathyroidal tumor extension, size, nodal involvement, distant metastases, technique of thyroid surgery and lymphatic dissection, initial treatment with 131Iodine, residual cervical disease, and histopathologic subtype. Surgical complications were correlated with the specific procedures completed on the thyroid gland or cervical lymphatics. RESULTS The overall progression-free survival rate was 67% (95%, CI: 61%-73%) at 10 years with 2 disease-related deaths. Regional lymph node and distant metastases were present in 74% and 25% of patients, respectively. Progression-free survival was less in younger patients (p = 0.009) and those with residual cervical disease after thyroid surgery (p = 0.001). Permanent hypocalcemia was more frequent after total or subtotal thyroidectomy (p = 0.001) while wound complications increased after radical neck dissections (p < 0.00001). CONCLUSIONS The progression-free survival rate was better after a complete resection and in older patients. Progression-free survival rate was the same after lobectomy or more extensive thyroid procedures, but comparison was confounded by the increased use of total or subtotal thyroidectomy in patients with advanced disease. The risk of permanent hypocalcemia increased when total or subtotal thyroidectomy was done. Thyroid lobectomy alone may be appropriate for patients with small localized lesions while total or subtotal thyroidectomy should be considered for more extensive tumors.

Life-threatening airway obstruction as a complication to the management of mediastinal masses in children

Life-threatening airway obstruction from large mediastinal masses in children poses a difficult diagnostic and therapeutic dilemma, requiring the close coordination of a pediatric surgeon, anesthesiologist, radiologist, and oncologist. To focus on this problem, the anesthetic and surgical management of 50 consecutive children with mediastinal masses treated between 1978 and 1984 were reviewed. Thirty children presented with respiratory symptoms; nine had life-threatening respiratory compromise with dyspnea, orthopnea, and stridor. Thirteen of these symptomatic children had marked compression of the trachea and/or mainstem bronchi on radiographic studies. The tracheal cross-sectional area which was measured by computed tomography was decreased by 35% to 93% of the normal tracheal dimensions in these children. Nonresectable malignant neoplasms including lymphoma, Hodgkins disease, rhabdomyosarcoma, and neuroblastoma were the eventual diagnoses in 10 of these patients. The other 3 patients were less than 4 years old and had benign lesions. General anesthesia was judged to be prohibitively risky in 5 of 13 patients. The diagnosis was established by node or needle biopsy under local anesthesia, and general anesthesia was deferred until the compromised airway was alleviated by radiation and chemotherapy. General anesthesia with endotracheal intubation was administered to 8 patients, 5 of whom developed total airway obstruction. Using a variety of maneuvers, ventilation was reestablished in all 5 patients.(ABSTRACT TRUNCATED AT 250 WORDS)

Congenital cystic lung disease: contemporary antenatal and postnatal management

Congenital cystic lung disease comprises a broad spectrum of rare but clinically significant developmental abnormalities, including congenital pulmonary adenomatoid malformations, bronchopulmonary sequestrations, bronchogenic cysts, and congenital lobar emphysema that result from perturbations in lung and airway embryogenesis. As congenital lung lesions are now more commonly recognized antenatally, mothers require accurate prenatal counseling and appropriate perinatal management. In light of long-term complications of infection and malignancy, there is growing consensus that infants with asymptomatic lesions should undergo elective excision of congenital pulmonary adenomatoid malformation (CPAM) or bronchopulmonary sequestration (BPS). This review will focus on advancements and current practice in the diagnosis and management of CPAM and BPS, identifying aspects of the literature that are confusing or controversial. Although our knowledge and pre- and postnatal management of lung lesions will continue to evolve and improve, there is a compelling need for a unified clinical and pathological classification system that creates a common platform for discussion, clinical management, and research.

Surgical repair of pectus excavatum markedly improves body image and perceived ability for physical activity: multicenter study.

OBJECTIVE. This study evaluated changes in both physical and psychosocial quality of life reported by the parent and child after surgical repair of pectus excavatum. METHODS. As part of a multicenter study of pectus excavatum, a previously validated tool called the Pectus Excavatum Evaluation Questionnaire was administered by the research coordinator, via telephone, to parents and patients (8–21 years of age) before and 1 year after surgery. Eleven North American childrens hospitals participated. From 2001 to 2006, 264 patients and 291 parents completed the initial questionnaire, and 247 patients and 274 parents completed the postoperative questionnaire. Responses used a Likert-type scale of 1 to 4, reflecting the extent or frequency of a particular experience, with higher values conveying less-desirable experience. RESULTS. Preoperative psychosocial functioning was unrelated to objective pectus excavatum severity (computed tomographic index). Patients and their parents reported significant positive postoperative changes. Improvements occurred in both physical and psychosocial functioning, including less social self-consciousness and a more-favorable body image. For children, the body image component improved from 2.30 ± 0.62 (mean ± SD) to 1.40 ± 0.42 after surgery and the physical difficulties component improved from 2.11 ± 0.82 to 1.37 ± 0.44. For the parent questionnaire, the childs emotional difficulties improved from 1.81 ± 0.70 to 1.24 ± 0.36, social self-consciousness improved from 2.86 ± 1.03 to 1.33 ± 0.68, and physical difficulties improved from 2.14 ± 0.75 to 1.32 ± 0.39. Ninety-seven percent of patients thought that surgery improved how their chest looked. CONCLUSIONS. Surgical repair of pectus excavatum can significantly improve the body image difficulties and limitations on physical activity experienced by patients. These results should prompt physicians to consider the physiologic and psychological implications of pectus excavatum just as they would any other physical deformity known to have such consequences.

Diagnosis, management, and outcome of cervicofacial teratomas in neonates: A Childrens Cancer Group study

The management of cervicofacial teratomas in neonates is often complicated and may result in significant morbidity and death. A Childrens Cancer Group (CCG) retrospective study was conducted to evaluate a multiinstitutional experience with the treatment of these extremely rare neoplasms. Twenty neonates with cervicofacial teratomas, presenting from 1971 to 1994, were identified from nine CCG institutions. Fourteen neonates had cervical teratomas, and six had orofacial teratomas. There were 12 males and eight females. A diagnostic prenatal ultrasound examination was performed in six cases. Life-threatening airway obstruction occurred in seven infants (35%) in the early postnatal period. Two neonates died in the delivery room without ever having their airway secured. Two other infants with a prenatal diagnosis survived only because tracheostomies were performed by pediatric surgeons who were in the delivery room. Three other patients were orally intubated, one after sustaining hypoxic cardiac arrest. Eighteen infants had their primary tumor excised. Three patients required tracheostomy. After resection, two patients had evidence of unilateral recurrent laryngeal nerve injury, and two required prolonged thyroid hormone replacement. Histological examination showed eight mature and seven immature teratomas. Four infants (20%) clearly had malignant lesions. Pulmonary metastases occurred in two patients and contributed to one late death at 6 months of age. The overall survival rate was 85%, and the mean follow-up period was 5 years (range, 2 months to 16 years). Twelve of 17 surviving patients (70%) have had an excellent functional and cosmetic outcome. Four children have varying degrees of developmental delay and mental retardation. Hypoxia at birth was believed to have contributed to these problems in two cases.(ABSTRACT TRUNCATED AT 250 WORDS)

Achalasia in childhood: A 20-year experience

The incidence of achalasia in children is lower than that in adults, but the disturbances in growth and development, and the severity of pulmonary symptoms are more profound. We have treated 20 children with the disease over the past 20 yr. All children were first treated with dilatation of the cardioesophageal junction either with filoform and followers, or in older children, pneumatic dilatation under fluoroscopic control. Five children, all older than 9 yr of age and all female, had prolonged relief of symptoms following two dilatations and never required a surgical procedure. Fifteen children responded unsatisfactorily to repeated dilatation and 12 of these accepted a surgical procedure. A modifier Heller procedure was performed in 11 children and one patient was treated with a transthoracic cardioplasty. The operations resulted in dramatic relief of symptoms, satisfactory weight gain, and the disappearance of pulmonary symptoms in every case. We recommend that all children under 9 yr of age should have a modified Heller procedure as primary therapy. In children older than 9 yr of age, dilatation is an appropriate initial therapy but should not be repeated unless significant improvement results.

Impact of bicycle helmet safety legislation on children admitted to a regional pediatric trauma center

PURPOSE The regional pediatric trauma center in Buffalo, NY, has been active in pediatric injury prevention programs, including community education and distribution of bicycle helmets, since 1990. Since June 1, 1994, the use of bicycle safety helmets for children under 14 years of age has been mandated by a state law in New York. The authors undertook this study to assess the impact of this legislation on the frequency of helmet use in children involved in bicycle crashes presenting to the regional pediatric trauma center, and to assess the impact of helmet use on the number and severity of head injuries. METHODS Bicycle crash victims (n = 208) admitted to a regional pediatric trauma center from 1993 to 1995 were studied retrospectively. Head injuries were classified as concussion alone, skull fractures, intracranial hemorrhages (ie, epidural, subdural, and subarachnoid), cerebral contusions, or diffuse cerebral edema alone (without any other intracranial injury). Helmeted children (HC) were compared with nonhelmeted children (NHC) using chi2 and Fishers Exact test. P value less than .05 was considered significant. RESULTS Only 31 children (15%) wore helmets at the time of the crash. Helmet use increased from 2%, during the period of education alone, to 26% after the legislation went into effect (P < .00001). The proportion of children suffering head injuries was similar in both groups (HC, 68%; NHC, 61%; P = NS). However, the type of head injury was different. HC were more likely to sustain concussion alone (HC, 65%; NHC, 44%; P < .03). HC were less likely to have skull fractures (HC, 0%; NHC, 13%; P < .02), and exhibited a trend toward less intracranial hemorrhages (HC, 0%; NHC, 9%; P = NS), cerebral contusions (HC, 3%; NHC, 5%; P = NS), and cerebral edema (HC, 0%; NHC, 0.6%; P = NS). Excluding the isolated concussions, head injuries were noted in only one HC, compared with 30 NHC (P < .04). None of the three children who died wore helmets at the time of the crash, and all died of multiple head injuries. CONCLUSIONS The bicycle helmet safety law resulted in a 13-fold increase in the use of bicycle helmets among the children admitted to a regional pediatric trauma center after bicycle crashes, but the helmet use remains inadequate. Helmet use reduced the severity of head injuries, and might have prevented deaths caused by head injuries.

Spectrum of hepatic hemangiomas: management and outcome.

PURPOSE Infants with multiple cutaneous hemangiomas often present with hepatic hemangiomas. They can follow a benign clinical course or require complex management. We reviewed our experience in the management of hepatic hemangiomas. METHODS We performed a retrospective review of patients (1996-2007) with hepatic hemangiomas treated in our institution. RESULTS Twenty-six patients were diagnosed with hepatic hemangiomas as follows: 8 focal, 12 multiple, and 6 diffuse lesions. Nineteen (73%) patients had associated cutaneous hemangiomas. Sixteen patients had multiple and 3 patients had single cutaneous hemangiomas. All patients with multiple or diffuse liver lesions were screened for heart failure and hypothyroidism. Congestive heart failure developed in 4 patients, 3/4 of these patients had diffuse lesions. Two patients required thyroid replacement because of elevated thyroid-stimulating hormone. Because of progression of disease, 9 patients required steroid treatment. Two patients were treated with vincristine and 3 patients received alpha-interferon because of poor response to steroid treatment. Two patients went on to surgical resection for failed response to medical management and worsening heart failure (left lobectomy, liver transplant). Both patients had uncomplicated postoperative courses. Five patients had a previously undescribed constellation of rapidly involuting cutaneous hemangiomas (gone by 3 months, glut-1-negative) with associated liver lesions also resolving at a faster pace (mean resolution of cutaneous hemangiomas, 1.9 vs 7.9 months; P = .001; liver, 5.8 vs 25.3 months; P = .004). All patients in our series survived. CONCLUSION Patients with multiple cutaneous hemangiomas should be screened for hepatic lesions. Patients with diffuse or multifocal liver hemangiomas should be screened for congestive heart failure and hypothyroidism. A subgroup of rapidly involuting cutaneous hemangiomas have a significantly shorter time for involution of hepatic lesions. The status of cutaneous lesions can be used as indicators for the liver hemangiomas.

Bladder pressure monitoring significantly enhances care of infants with abdominal wall defects: A prospective clinical study

Increased intraabdominal pressure (IAP) has been demonstrated to cause intestinal and renal ischemia in both animals and humans. Neonates undergoing closure of anterior abdominal wall defects are at risk for these complications from markedly increased IAP, which are putatively responsible for a 13% to 20% mortality. In an effort to decrease morbidity and mortality we performed a 4-year prospective clinical study to determine if monitoring IAP using bladder pressure (BdP) measurements would significantly improve perioperative care in infants with abdominal wall defects. Forty-two consecutive infants with gastroschisis (28) and omphalocele (14) were prospectively studied. Intraoperative and serial postoperative measurements of BdP were obtained from an indwelling bladder catheter using a standard pressure transducer. Methods of initial closure, as well as manipulations in sedation, paralysis, and silo reduction, were selected to keep BdP < 20 mm Hg. Bladder pressure monitoring significantly altered the management of 64% of our patients, particularly those with gastroschisis (74%). Thirteen patients with gastroschisis underwent staged closure; in 7 (54%) this decision was based on high BdP even though bowel reduction was mechanically possible. Elevated BdP influenced the closure method and timing of silo reductions in 5 of 14 (42%) infants with omphalocele. There were no episodes of renal failure or refractory oliguria. There were three patients in a single cluster who developed uncomplicated, nonsurgical necrotizing enterocolitis late in their respective courses. One patient whose bowel was placed in a silo had severe hypotension associated with group B streptococcal sepsis and subsequently developed necrotic bowel despite low BdP.(ABSTRACT TRUNCATED AT 250 WORDS)