Roshni Dasgupta

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.

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.

Update on rhabdomyosarcoma

Rhabdomyosarcoma (RMS) is a malignant childhood tumor of mesenchymal origin that currently has a greater than 70% overall 5-year survival. Multimodality treatment is determined by risk stratification according to pretreatment stage, postoperative group, histology, and site of the primary tumor. Pretreatment staging is dependent on primary tumor site, size, regional lymph node status, and presence of metastases. Unique to RMS is the concept of postoperative clinical grouping that assesses the completeness of disease resection and takes into account lymph node evaluation. At all tumor sites, the clinical grouping, and therefore completeness of resection, is an independent predictor of outcome. Overall, the prognosis for RMS is dependent on primary tumor site, patient age, completeness of resection, extent of disease, including the presence and number of metastatic sites and histology and biology of the tumor cells. Therefore, the surgeon plays a vital role in RMS by contributing to risk stratification for treatment, local control of the primary tumor, and outcome. The current state-of-the-art treatment is determined by treatment protocols developed by the Soft Tissue Sarcoma Committee of the childrens Oncology Group.

Close distal margin and rectal cancer recurrence after sphincter-preserving rectal resection.

PURPOSE: Negative surgical margins are important for local control of rectal cancer treated with sphincter-preserving surgery. However, the association of rectal cancer recurrence with close distal margin is not well established. METHODS: Data were extracted from a prospective database of patients collected between 1991 and 2003. Included were 627 patients who underwent curative low anterior resection with total mesorectal excision for rectal cancer 2 to 12 cm from the anal verge. Three hundred ninety-nine patients received neoadjuvant therapy, 65 received postoperative adjuvant therapy alone, and 163 were treated with surgery alone. Median follow-up was 5.8 years. RESULTS: On multivariable analysis, overall recurrence was associated with pathologic stage, lymphovascular invasion, and distal margin. Mucosal recurrence was uncommon; only 16 events were recorded, and of those only 8 were at the initial site of isolated tumor recurrence; 7 of the 8 were surgically salvaged. On univariable analysis, mucosal recurrence was associated with close distal margin (5 vs 2% at 5 y) and lymphovascular invasion (7 vs 2%). Pelvic recurrence, other than isolated mucosal recurrence, was associated with distal location (6 vs 4% at 5 y) and lymphovascular invasion (11 vs 4%). Distal margin as a continuous variable was associated with overall recurrence (excluding isolated mucosal recurrence). CONCLUSIONS: Close distal resection margin identifies patients with increased risk of mucosal and overall cancer recurrence. Although neither causality nor a minimally acceptable margin length can be defined, the data support the importance of achieving a clear distal resection margin in the surgical management of rectal cancer.

Patterns of Chemotherapy-Induced Toxicities in Younger Children and Adolescents with Rhabdomyosarcoma: A Report from the Children’s Oncology Group Soft Tissue Sarcoma Committee

Patients aged >10 years with rhabdomyosarcoma have an inferior outcome compared with patients ages 1 to 9 years, which may be explained by toxicities (adverse events [AEs]) that result in chemotherapy dose reductions.

Management of congenital diaphragmatic hernia: A systematic review from the APSA outcomes and evidence based practice committee

OBJECTIVE Variable management practices complicate the identification of optimal strategies for infants with congenital diaphragmatic hernia (CDH). This review critically appraises the available evidence to provide recommendations. METHODS Six questions regarding CDH management were generated. English language articles published between 1980 and 2014 were compiled after searching Medline, Cochrane, Embase and Web of Science. Given the paucity of literature on the subject, all studies irrespective of their rank in the levels of evidence hierarchy were included. RESULTS Gentle ventilation with permissive hypercapnia provides the best outcomes. Initial high frequency ventilation may be considered but its overall efficacy is unproven. Routine inhaled nitric oxide (iNO) or other medical adjuncts for acute, severe pulmonary hypertension demonstrate no benefit. Evidence does not support routine administration of pre- or postnatal glucocorticoids. Mode of extracorporeal membrane oxygenation (ECMO) has little bearing on outcomes. While the overall timing of repair does not impact outcomes, early repair on ECMO has benefits. Open repair leads to significantly fewer recurrences. Polytetrafluoroethylene (PTFE) is the most durable patch repair material. CONCLUSIONS Limited high-level evidence prevents the development of robust management guidelines for CDH. Prospective, multi-institutional studies are needed to identify best practices and optimize outcomes.

Asymptomatic malrotation: Diagnosis and surgical management An American Pediatric Surgical Association outcomes and evidence based practice committee systematic review

OBJECTIVE Patients with malrotation, or an intestinal rotation abnormality (IRA), can experience serious adverse events. Increasingly, asymptomatic patients are being diagnosed with malrotation incidentally. Patients with symptomatic malrotation require surgery in an urgent or semiurgent manner to address their symptoms. The treatment of asymptomatic or incidentally discovered malrotation remains controversial. METHODS Data were compiled from a broad search of Medline, Cochrane, Embase and Web of Science from January 1980 through January 2013 for five questions regarding asymptomatic malrotation. RESULTS There is minimal evidence to support screening asymptomatic patients. Consideration may be given to operate on asymptomatic patients who are younger in age, while observation may be appropriate in the older patient. If reliably diagnosed, atypical malrotation with a broad-based mesentery and malposition of the duodenum can be observed. Regarding diagnostic imaging, the standard of care for diagnosis remains the upper gastrointestinal contrast study (UGI), ultrasound may be useful for screening. A laparoscopic approach is safe for diagnosis and treatment of rotational abnormalities. Laparoscopy can aid in determining whether a patient has true malrotation with a narrow mesenteric stalk, has nonrotation and minimal risk for volvulus, or has atypical anatomy with malposition of the duodenum. It is reasonable to delay Ladd procedures until after palliation on patients with severe congenital heart disease. Observation can be considered with extensive education for family and caregivers and close clinical follow-up. CONCLUSIONS There is a lack of quality data to guide the management of patients with asymptomatic malrotation. Multicenter and prospective data should be collected to better assess the risk profile for this complex group of patients. A multidisciplinary approach involving surgery, cardiology, critical care and the patients caregivers can help guide a watchful waiting management plan in individual cases.

Congenital adrenal hyperplasia: surgical considerations required to repair a 46,XX patient raised as a boy

21-hydroxylase deficiency (P450 CYP21) accounts for 90% of cases of congenital adrenal hyperplasia (CAH), which is associated with abnormally low cortisol and high production of androgen precursors and is the most common cause of ambiguous genitalia. Increased androgen causes in utero virilization of the fetus, consisting of clitoral enlargement, an urogenital sinus, and labioscrotal enlargement and fusion. This is the first case in an experience covering more than 30 years, of a 46,XX patient raised as a boy. The authors report a case of a Pakistani patient born of a consanguineous union, who came to medical attention at age 3 because of severe genital ambiguity; genetic analyses showed that the child was a compound heterozygote for CAH. The surgical management of this patient consisted of (1) staged hypospadias repairs preceded by testosterone therapy, (2) creation of a bladder graft neourethra, (3) removal of müllerian structures, (4) correction of bifid prepenile scrotum, and (5) insertion of testicular prostheses. The commitment to raise a 46,XX child as a boy is a very rare event. With a series of staged complex surgical procedures and careful steroid replacement, normal secondary sexual characteristics can be achieved in these children.

Response of Blue Rubber Bleb Nevus Syndrome to Sirolimus Treatment.

Blue rubber bleb nevus syndrome (BRBNS) is a rare multifocal venous malformation syndrome involving predominantly the skin and gastrointestinal tract. Traditional treatment modalities include corticosteroids, interferon‐α, sclerotherapy, and aggressive surgical resection. Sirolimus has been used in several single case reports.

Lung nodules in pediatric oncology patients: a prediction rule for when to biopsy

PURPOSE The purpose of the study was to develop a prediction rule regarding the factors that most accurately predict the diagnosis of a malignancy in a lung nodule in the pediatric oncology patient. METHODS A retrospective review of pediatric oncology patients that underwent lung nodule resection between 1998 and 2007 was performed. Multivariable logistic regression was used to create a prediction rule. RESULTS Fifty pediatric oncology patients underwent 21 thoracotomies and 48 thoracoscopies to resect discrete lung nodules seen on computed tomographic scans during workup for metastasis or routine surveillance. The mean nodule size was 10.43 ± 7.08 mm. The most significant predictors of malignancy in a nodule were peripheral location (odds ratio [OR], 9.1); size between 5 and 10 mm (OR, 2.78); location within the right lower lobe (OR, 2.43); and patients with osteosarcoma (OR, 10.8), Ewing sarcoma (OR, 3.05), or hepatocellular carcinoma (OR, 2.38). CONCLUSIONS Lesions that are between 5 and 10 mm in size and peripherally located in patients with osteosarcoma, Ewing sarcoma, or hepatocellular carcinoma are most likely to be malignant. Use of a prediction rule can help guide clinical practice by determining which patients should undergo surgical resection of lung nodules and which patients may be closely observed with continued radiologic studies.