Liver, pancreas and kidney transplantation for the treatment of Wolcott-Rallison syndrome.

We present the case of a child who underwent a combined liver, pancreas and double kidney transplant following complications of Wolcott-Rallison syndrome (WRS) a rare genetic disorder that causes infantile insulin-dependent diabetes mellitus (IDDM) and often death in childhood from fulminant liver and concomitant kidney failure. WRS is characterized clinically through infantile IDDM, propensity for liver failure following viral infections, bone dysplasia and growth failure and developmental delay. Fewer than 60 cases with WRS are reported in the literature, mostly from consanguineous parents. Future episodes of liver failure, the main contributor to the increased mortality in WRS, may be prevented through timely liver transplantation. To the best of our knowledge, transplantation has not been utilized to manage complications of WRS prior to this report.

Surgery-associated complications in necrotizing enterocolitis: A multiinstitutional study.

PURPOSE: This study was designed to evaluate the wound and stomal complication rate associated with surgical intervention in infants with necrotizing enterocolitis (NEC). METHODS: Comprehensive demographic and perioperative data were collected prospectively from 4 separate university hospitals on 51 infants with surgically treated NEC. The postoperative complication rate included wound (infection, dehiscence) and stomal (prolapse, retraction, necrosis, stricture) problems. For analysis, patients were grouped based on gestational age less than 28 weeks (group I, n = 30) and >/=28 weeks (group II, n = 21). Z-score analysis was used for intergroup evaluation. RESULTS: Significantly more infants in group I (21 of 30 [70%] versus group II, 6 of 21 [29%]; P <.001) were treated initially with Penrose drainage alone, but most eventually underwent laparotomy (group I, 28 of 30 [93%] versus group II, 19 of 21 [91%]; P value, not significant). The combined stomal/wound complication rate was significantly higher in group I (14 of 30 [47%]) versus group II (6 of 21 [29%]; P <.025). Of 51 patients, one operation was required in 23 (45%), 2 in 18 (35%), 3 in 8 (16%), and 4 in 2 (4%). CONCLUSIONS: Although the stomal/wound complication rate was significantly higher in group I, both groups had very substantial complication rates, emphasizing the vulnerability of this infant population. Parents, especially of very premature babies, should be advised that multiple operations are likely and that complications should be expected.

The role of surgery in children with neurofibromatosis.

BACKGROUND/PURPOSE: Neurofibromatosis frequently is complicated by the development of symptomatic lesions such as optic gliomas and plexiform neurofibromas that require operative resection. Although characteristically benign, these neoplasms have often devastating functional and cosmetic effects and must be monitored for malignant transformation. The purpose of this study is to identify and describe the surgical considerations in the care of children with neurofibromatosis. METHODS: The authors reviewed the charts of all children (<21) at our institution with neurofibromatosis who underwent an operative procedure from 1979 to 1999. Patient demographics, symptomatic lesions, malignant transformation, form of surgical intervention, type of anesthesia, and outcome were collected. RESULTS: A total of 249 patients with either neurofibromatosis 1 or 2 were identified. Of these, 50 (20%) underwent a total of 93 operations. The average age at operation was 9.4 years (1.2 to 21 years). There were 40 soft tissue procedures, 21 intracranial, and 32 miscellaneous. The soft tissue masses typically were treated with wide local excision, and in 8 of these procedures multiple resections were performed. Fourteen of the 50 patients had malignancies. Five of the tumors were soft tissue sarcomas, and 9 were intracranial malignancies. Three patients died, 2 from malignancy and 1 from acute, obstructive hydrocephalus after operation. There were 3 patients alive with malignancy and 8 others living with varying levels of disability. CONCLUSIONS: Neurofibromatosis in the pediatric patient frequently requires surgical intervention, often because of symptoms such as pain or cosmetic deformity, or for malignancy. Children should be watched carefully for signs of malignant transformation and undergo biopsy for neurofibromas that exhibit rapid growth. Management of sarcomas should be aggressive with consideration given to re-excision, placement of brachytherapy catheters, metastectomy, and limb salvage with adjuvant therapy when possible. Preoperatively, children should receive clinical and radiographic (computed tomography or magnetic resonance imaging) evaluation for hydrocephalus.

Multidisciplinary management of pediatric soft-tissue sarcoma.

The management of pediatric soft-tissue sarcomas has improved drastically through the use of multimodal therapy. These tumors include rhabdomyosarcomas and nonrhabdomyosarcomas. Both are staged using physical, radiographic, and histologic evaluation, and both have intricate staging and grouping systems that correlate closely with prognosis. However, approaches to therapy for the two tumor types remain somewhat different. Rhabdomyosarcomas are treated primarily with chemotherapy. Surgical intervention is limited to initial biopsy, wide local excision when clear margins are feasible, and resection of residual disease. Radiation therapy is reserved for patients with persistent or recurrent disease and may be delivered by external beam or brachytherapy. Nonrhabdomyosarcomas are best treated primarily by surgical resection, although radiation and chemotherapy are now being used with some success. Another major difference concerns evaluation of lymphatics. Nonrhabdomyosarcomas in children frequently behave similarly to adult sarcomas, and less commonly involve regional lymph nodes, whereas pediatric patients with rhabdomyosarcomas often have nodal involvement necessitating surgical evaluation of regional lymph nodes as part of the staging protocol. Multimodal therapy has led to improved survival as well as better functional and cosmetic results. With further clinical trials and improved techniques such as brachytherapy and lymphatic mapping with sentinel node biopsy, we expect to continue to optimize therapy for pediatric patients with soft-tissue sarcomas.

Wilms' tumor. Overview of National Wilms' Tumor Study Group results.

Since its inception in 1969, the NWTSG has performed successful studies that are now the model for the management of pediatric malignancies. Future studies may use genetic markers to stratify high-risk patients beyond the traditional staging system. Therapy will continue to be evaluated to determine the minimal therapy necessary to achieve the best outcome for children with Wilms' tumor.

Lymphatic mapping with sentinel node biopsy in pediatric patients.

BACKGROUND/PURPOSE: Lymphatic mapping with sentinel node biopsy is used widely in adult melanoma and breast cancer to determine nodal status without the morbidity associated with elective lymph node dissection. This technique can be used in children to determine lymph node status with limited dissection and accurate interpretation. The authors report their initial experience. METHODS: The charts of patients who underwent lymphatic mapping with sentinel node biopsy were reviewed retrospectively. Lymphoscintigraphy was performed in patients with truncal lesions 24 hours before surgery to determine the draining nodal basin (for surgical mapping). The tumors were injected 1 hour preoperatively with technetium sulfur colloid and in the operating room with Lymphazurin blue. The draining basin was examined using a radioisotope detector. The blue nodes with high counts were localized and removed. If nodal metastases were identified, lymph node dissection was recommended. Four patients were injected only with Lymphazurin blue. RESULTS: Thirteen children (7 girls, 6 boys; mean age, 7 years) underwent lymphatic mapping with sentinel node biopsy. The tumor types were as follows: 8 malignant melanoma (6 extremity, 2 truncal), 1 malignant peripheral nerve sheath tumor, 1 alveolar soft part sarcoma, and 3 rhabdomyosarcoma. A mean of 2.4 nodes (range, 1 to 6) were removed from each patient. Six patients had a positive sentinel node. Formal lymph node dissection was performed on 4 of the 6 patients, 1 of whom had further nodal disease with 2 of 13 nodes containing micrometastases. One of the 6 patients refused lymph node dissection and adjuvant therapy; the final patient had rhabdomyosarcoma, a malignancy for which lymph node dissection is not indicated. Pulmonary metastasis developed 26 months after diagnoses in the patient with alveolar soft part sarcoma and a negative sentinel node. This patient was injected only with Lymphazurin blue at the time of sentinel node biopsy and refused adjuvant therapy. There have been no other recurrences. There were no complications related to lymphatic mapping or sentinel node biopsy. CONCLUSIONS: Lymphatic mapping with sentinel node biopsy, using both technetium-labeled sulfur colloid and Lymphazurin blue, can be performed safely in pediatric skin and soft tissue malignancies. Further study with long-term follow-up will determine the utility and accuracy of this technique in pediatric malignancies.

Emergent abdominal decompression with patch abdominoplasty in the pediatric patient.

BACKGROUND/PURPOSE: Abdominal compartment syndrome (ACS) is the cardiac, pulmonary, and renal dysfunction that occurs as a result of elevated intraabdominal pressure. The authors present their experience with patch abdominoplasty (PA) in pediatric patients as a means to treat and prevent ACS. METHODS: The charts of patients who underwent PA were reviewed retrospectively. ACS was defined as the increased oxygen requirements and elevation of peak inspiratory pressures (PIP) associated with abdominal distension and worsening renal and or cardiac function. RESULTS: A total of 23 patients (13 boys) were treated (average age, 23 months). Diagnoses included necrotizing enterocolitis (NEC, n = 13), trauma (n = 3), Hirschsprung's enterocolitis (n = 2), perforated bowel (n = 4), and bilateral Wilms' tumor with bowel obstruction (n = 1). Oxygen requirements decreased after patch abdominoplasty (mean preoperative FIO2, 0.87 +/- 24, mean postoperative, 0.67 +/- 24 [P = .01]). The PIP decreased significantly in the 13 patients who survived (mean preoperative PIP, 33 +/- 8, mean postoperative PIP, 27 +/- 7 [P = .01]). These PIPs failed to respond in the 8 nonsurvivors (mean preoperative PIP, 35 +/- 10, mean postoperative PIP, 33 +/- 14 [P value not significant]). Six of the 8 nonsurvivors had NEC. Complications of intraabdominal abscess and enterocutaneous fistula were seen in 5 patients, all of who had NEC. CONCLUSIONS: Patch abdominoplasty effectively decreases airway pressures and oxygen requirements associated with ACS. Complications with PA occur primarily in patients with NEC. Failure to respond with a decrease in PIP and FIO2 requirements is an ominous sign.

Preoperative staging, prognostic factors, and outcome for extremity rhabdomyosarcoma: a preliminary report from the Intergroup Rhabdomyosarcoma Study IV (1991-1997).

BACKGROUND: During the fourth Intergroup Rhabdomyosarcoma (RMS) Study (IRS IV, 1991-97), a preoperative staging system was evaluated prospectively for the first time. The authors evaluated this staging system and the role of surgery in extremity RMS in contemporary multimodal therapy. METHODS: A total of 139 patients (71 girls; median age, 6 years) were entered in IRS IV with extremity-site RMS. Stage was assigned by the IRSG Preoperative Staging System. Postsurgical group was determined by tumor status after initial surgical intervention. Multivariate analysis was performed using all pretreatment factors that were significant by univariate analysis, including clinical Group (i.e., I through IV), tumor invasiveness (T1,T2), nodal status (N0,N1), and tumor size (< or > or =5 cm). Failure-free survival rates (FFS) and survival rates were estimated using the Kaplan and Meier method. RESULTS: Preoperative staging and clinical group distribution were as follows: Stage 2, n = 34; Stage 3, n = 73; Stage 4, n = 32; Group I, n = 31; Group II, n = 21; Group III, n = 54; Group IV, n = 33. Three-year FFS was 55%, and the overall survival rate was 70%. Eighty-seven patients had either unresectable, gross residual disease (Group III) or metastases (Group IV). FFS was significantly worse for these patients with advanced disease, compared with that for patients with complete resection or with only microscopic residual tumor (i.e., Group I or II; Group I, 3-year FFS, 91%; Group II, 72%; Group III, 50%; Group IV, 23%; P<.001). Lymph nodes were evaluated surgically in 76 patients with positive results in 38. Clinically, 13 additional patients had nodal disease. Both stage and group were highly predictive of outcome and were highly correlated. By multivariate analysis, none of the other variables were predictors of FFS. CONCLUSIONS: This review confirms the utility of pretreatment staging for stratification of patients with extremity RMS with widely different risks of relapse, thereby paving the way for development of risk-based therapy. Group (operative staging) remains the most important predictor of FFS, emphasizing the importance of complete gross resection at initial surgical intervention, when feasible without loss of limb function. The high incidence of nodal disease in the patients who had lymph node biopsy confirms the need for surgical evaluation of lymph nodes to ensure accurate staging in children with extremity rhabdomyosarcoma.