Louise Schnaufer

A review of 17 IV-S neuroblastoma patients at the children's hospital of Philadelphia

The records of 207 neuroblastoma patients seen at the Childrens Hospital of Philadelphia between 1944 and 1977 were reviewed to study some of the features associated with the unusually good prognosis found in patients with Stage IV‐S neuroblastoma. Initially, 22 patients appeared to fit the criteria of small primary tumor and distant disease in liver, skin, and/or marrow without evidence of bone metastases; 5 patients were subsequently rejected as being incorrectly staged. The remaining 17 patients had abdominal primary tumors and hepatic disease; in 12 of the 17, an enlarged liver was the presenting sign. Six patients had skin lesions, 4 had disease in the marrow on routine smear, and additional sites of spread were pancreas and bowel serosa. The treatment given was not systematic, and it was not possible to correlate any specific form of therapy with a satisfactory outcome. Eleven of 17 patients survived; 6 of 11 survivors had spontaneous regression of all or part of their disease, 5 of 6 who died received irradiation, chemotherapy, or both. Death usually occurred in the first month as a complication of the local disease; 1 patient succumbed to radiation nephritis. This study establishes that the special pattern of widespread neuroblastoma termed Stage IV‐S does exist, and that it is associated with a good prognosis. Careful consideration should be given before selecting treatment for the Stage IV‐S child because spontaneous regression is likely to occur in most of them. In patients with rapidly enlarging livers, renal or pulmonary complications may develop because of liver bulk or coagulopathies. Treatment should be directed to the liver in these cases because distant metastases seldom supervene. Low‐dose irradiation, mild chemotherapy, and possibly surgical release of intraabdominal pressure using a silastic patch have all been effective. Unfortunately, patients occasionally succumb to local disease in spite of these and more aggressive measures.

Congenital hyperinsulinism and the surgeon: Lessons learned over 35 years

BACKGROUND/PURPOSEnCongenital hyperinsulinism induces severe and unremitting hypoglycemia in newborns and infants. If poorly controlled, seizures and irreversible brain damage may result. Subtotal (<95%) or near-total (95% to 98%) pancreatectomy have been performed for glycemic control in babies who do not respond to aggressive medical therapy. Because hypoglycemia often persists after subtotal resection, 95% pancreatectomy has emerged as the procedure of choice. To define the effect of more or less extensive pancreatectomy on the management and outcome of refractory congenital hyperinsulinism, the authors examined our single institutional experience.nnnMETHODSnThe records of children treated between 1963 and 1998 for congenital hyperinsulinism, and who required pancreatectomy, were reviewed. Outcome parameters included glycemic response to surgery, need for reresection, surgical morbidity, surgical and long-term mortality, and development of diabetes mellitus (DM). A complete response was defined as discharge to home on no glycemic medications, no continuous feedings, and without DM. Histological reports were reviewed and categorized as either diffuse or focal disease.nnnRESULTSnOf 101 children treated for congenital hyperinsulinism during this period, 53 (50%) required pancreatectomy for glucose control. Mean follow-up for the study population was 9.8 +/- 1.1 years. Overall, 23 children (43%) showed a complete response, occurring in 50% of patients having > or = 95% pancreatectomy (n = 34), but in only 19% having less than 95% resection (n = 16). The remaining three babies had local excision of a solitary focal lesion, and each showed a complete response. Histopathology showed diffuse islet abnormalities in 42 specimens (79%) and solitary focal lesions in 11 (21%). A complete response was observed for 82% of focal but only 33% of diffuse lesions. Eight patients (15%) required reresection for persistent hypoglycemia, seven having diffuse lesions and one focal. Surgical morbidity occurred in 13 cases (26%), and the 30-day surgical mortality rate was 6%, each death (n = 3) occurring before 1975. DM developed in seven children (14%), each having diffuse lesions, and was independent of resection type.nnnCONCLUSIONnBecause euglycemia is more readily restored, and because the risks for surgical complications and DM do not appear increased, the authors recommend 95% pancreatectomy as the initial procedure of choice for newborns and infants with congenital hyperinsulinism.

Esophageal Replacement Using the Colon: Is It a Good Choice?

Thirty-eight children (2 months to 26 years of age) underwent esophageal replacement at our institution between 1962 and 1993. Twenty-four patients had esophageal atresia, with the replacement performed at a mean age of 17 months. The remaining patients (37%) had strictures and were older (mean, 7.4 years). Replacement procedures involved the right colon in 61% of cases and the transverse left colon in the others (39%). Sixty-three percent were placed substernally and 37% were done in transthoracic fashion. The average length of stay in the hospital was 34 days (range, 11 to 256 days.) Early complications (within 30 days) included cervical anastomotic leaks (11 patients; 29%) pneumonia (4), would infection (2), pneumothorax/hemothorax (3), wound dehiscence (1), prolonged ventilation (2), vocal cord paralysis (1), Horners syndrome (1), pancreatitis (1), and perforated graft (1). Despite the incidence of early leaks, only two persisted long-term (more than 3 months). Other late complications included significant proximal strictures (5), and cologastric strictures developed in five patients. Seven cases were considered graft failures (18%), and all of these eventually require graft replacement. Additional problems included redundant graft requiring revision (4) and dumping syndrome (2). There were six cases of intestinal obstruction caused by adhesions. Four of these involved intrathoracic obstruction of the graft and two involved small bowel obstruction. There was only one death, which occurred late and was not related to the primary disease or procedure. Long-term follow-up data were available for 20 patients (53%). The follow-up period ranged from 1 to 33 years (mean, 12 years). Fourteen had excellent results after the initial interposition, being able to eat and function well without any further intervention. Seven patients (18%) have had poor results and 17 (45%) required additional procedures to obtain good functional results. In our experience, the colon continues to be a good option for esophageal replacement, but additional procedures frequently are necessary to optimize the functional outcome. Good results can be expected in the majority of cases, but late problems (ie, redundant colon and poor emptying) are not unusual, and careful follow-up is essential in the management of such patients.

Surgical Manifestations of Eosinophilic Gastroenteritis in the Pediatric Patient

Eosinophilic gastroenteritis, an inflammatory bowel disease of unknown etiology, occurs in one of 10,000 admissions to this Childrens Hospital. We had added 4 cases to the 11 retrieved from a literature review. The inflammation is characterized by mature eosinophils predominating a transmural process which may produce pain, obstruction, perforation, bleeding, or fistulae. All levels of the gastrointestinal tract are involved, but stomach (25.9%) and small bowel (66.7%) lesions are most common. Eosinophilia occurs in 61% of children and allergy in 13%. X-rays may demonstrate a diffuse or localized process. Operative intervention may be necessary to exclude tumors or regional enteritis, and at times to extirpate complications of local disease, but conservative therapy is the treatment of choice for this exacerbating-remitting disease.

Treatment strategies for infants with malignant sacrococcygeal teratoma

Twelve children with malignant sacrococcygeal teratoma containing embryonal carcinoma, were treated at the Childrens Hospital of Philadelphia between 1971 and 1980. Their ages at diagnosis ranged from 2 days to 23 mo; 8 of the 12 (67%) were girls. Five of the 12 patients presented with localized tumors which were grossly completely excised. Four received no further therapy, and all 4 recurred with histologically documented embryonal carcinoma. Despite subsequent treatment with radiation therapy (RT) and chemotherapy (vincristine, actinomycin D, and cyclophosphamide, collectively called VAC, in all 4 plus adriamycin in 3, only 1 has survived free of disease, 8 yr from diagnosis. The other 3 died of tumor (2) or pneumonia after pulmonary RT (1). The fifth patient in this group received VAC with adriamycin (total dose, 350 mg/sq m) and died of autopsy-proven cardiotoxicity without tumor. The remaining 7 patients presented with either unresectable local tumor (3) or distant metastases (4). One of these 7 died 6 days after biopsy with massive liver metastases. The other 6 children were treated after operation with VAC (5) or vincristine and actinomycin D (1) chemotherapy, and 5 also received RT to the pelvis. One has survived free of disease at 6 yr after treatment with VAC and adriamycin and pelvic RT, but the other 5 died of tumor (2) or of pneumonia after pulmonary RT (3). Surgery alone is inadequate for successful management of children with malignant sacrococcygeal teratoma. Chemotherapy with VAC, with or without adriamycin, can be effective when combined with radiation, but fatal pulmonary toxicity can result from such combined therapy. The optimal therapeutic program for children with malignant sacrococcygeal teratoma is still evolving.

Meconium Hydrocele in a Female Newborn: An Unusual Cause of a Labial Mass

Meconium peritonitis results from in utero perforation of the bowel and subsequent spillage of meconium into the peritoneal cavity. Free communication of the peritoneal space with the processus vaginalis during gestation permits formation of a meconium hydrocele. Meconium hydrocele has been reported in the newborn scrotum but to our knowledge there has been no previous report of meconium hydrocele in the labium of a female neonate. The predominance of meconium hydrocele in the male infant may be due to the obliteration of the processus vaginalis occurring later in the male than in the female fetus.

Gastric Duplication Presenting as Cyclic Abdominal Pain

number of its causes are overwhelming. A careful history and study of the associated signs and symptoms along with physical and laboratory findings, can often identify the underlying cause. Nevertheless, recurrent abdominal pain of a cyclic nature in an otherwise well three-year-old can be difficult to define under the best of circumstances, and prompts this report of an unusual instance presenting without any associated findings.

Extraskeletal osteogenic sarcoma after treatment for Wilms' tumor.

A large proportion of children with Wilms tumor will become long‐term disease‐free survivors. A small number of these children are at risk of developing second malignant neoplasms. There have been no previous reports of osteogenic sarcoma of the chest wall following treatment of Wilms tumor. Our patient was age seven years when he received surgery, radiation therapy and chemotherapy for Wilms tumor, eight years when he received radiation and chemotherapy for pulmonary metastases of Wilms tumor, and 13 years when he developed osteogenic sarcoma of the chest wall. Cancer 50:1894‐1898, 1982.

Nonsurgical Management of Children With Recurrent OT Unresectable Fibromatosis

At The Childrens Hospital of Philadelphia, since 1971, six children 3 months to 17 years of age with fibromatosis have been treated with a combination of vincristine, actinomycin D, and cyclophosphamide (VAC). The first three patients also received radiation therapy (5,500 rads). Locally recurrent tumors developed in four of the children after previous operative removal; the other two had tumors that could not be removed initially. The tumors arose in the neck (three patients), pelvis (two patients), or foot (one patient). In the three patients treated with VAC alone, complete disappearance of tumor was confirmed at second operation in two, and greater than 75% shrinkage on CT scans occurred in the third, all at 4 to 6 months after VAC was started. In two of the three patients who received VAC plus radiation therapy, complete disappearance of tumor occurred at 13 and 16 months; the third had no response. Five of the six patients are free of recurrent fibromatosis at 1, 2, 4, and 11 years after VAC was begun; the sixth has required multiple operations during the last 6 years. We conclude that combination chemotherapy with VAC can produce regression of fibromatosis in some children with recurrent or unresectable lesions. The administration of VAC should be considered for children with fibromatosis in whom operative removal is not feasible, would prove mutilating, or is unlikely to produce long-term control of the disease.

Hirschsprung’s Disease

Hirschsprungs disease or aganglionic megacolon is an anomaly caused by the absence of ganglion cells in the myenteric plexus of the distal colon. It produces intestinal obstruction or lethal enterocolitis in the neonatal period and constipation of varying degree in the older child. The diagnosis can be made by history alone and confirmed by physical findings, barium enema, motility studies, and rectal biopsy. Colostomy may be a life saving measure in the newborn, to be followed by a definitive pull-through procedure before the age of 1 year. Operative correction consists of various techniques all of which aim for excision of the aganglionic segment with preservation of the internal anal sphincter. The major pitfall of these procedures is a too perfectly preserved sphincter which remains spastic and still produces obstruction. Some damage to this muscle must be accomplished either during the procedure or postoperatively by bouginage in order to obtain a satisfactory result.