[Show abstract][Hide abstract] ABSTRACT: Intracranial germ cell tumors (GCTs) are not a common disease. We reviewed the experience of a single institution to determine the variables that affect treatment outcome.
A retrospective review of patients with the diagnosis of intracranial germ cell tumors treated in a single institution (KFSHRC) during the period from March 1985 to December 2007.
Fifty-seven patients with the diagnosis of intracranial GCT were recorded in the KFSHRC Tumor Registry during the period from 1985 to 2007. Seven patients with a pineal region tumor treated as germinomas in the earlier years without a tissue diagnosis were excluded. This retrospective study was restricted to the remaining 50 patients with a tissue or marker diagnosis: 31 germinomas and 19 non-germinomatous germ cell tumors (NGGCTs).
The 10-year overall survival (OS), event-free survival (EFS) and relapse-free survival (RFS) were 87%, 88% and 96% for patients with germinoma, with a median follow-up of 4.5 (range 2-17) years, compared with 26%, 29% and 46% for patients with NGGCT with a median follow-up of 3 (range 1.5-13) years. For NGGCT, variables favorably influencing OS were younger age (< 16 y vs ≥16 y, P=.01), higher tumor dose (>50 Gy vs ≤50 Gy; P=.03) and later year of diagnosis (>1990 vs <1990 P=.002).
Tissue diagnosis of GCTs is mandatory prior to treatment except for patients with elevated markers. In germinoma, localized radiotherapy (RT) for M0 patients may be adequate. Long-term follow-up is needed to define the benefit of adding chemotherapy. For NGGCT, the use of combined modality treatment and RT dose ;gt;50 Gy are important factors that influence the outcome. Second-look surgery and resection of residual/ refractory tumors is always recommended.
Annals of Saudi medicine 07/2012; 32(4):359-65. DOI:10.5144/0256-4947.2012.359 · 0.49 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Reduced-intensity protocols for pediatric Hodgkin's lymphoma are aimed at preserving excellent relapse-free survival while decreasing the incidence of late effects.
We retrospectively reviewed the outcome of 123 children treated consecutively for Hodgkin's lymphoma at a single institution. Patients with stages I-IIIB disease received three cycles of mechlorethamine, vincristine, procarbazine, and prednisone (MOPP)/ doxorubicin, bleomycin, and vinblastine (ABV) followed by 15 Gy of extended-field irradiation, while those with stage IV disease were treated with six to eight cycles of MOPP/ABV chemotherapy with or without radiotherapy.
At a median follow-up of 8.5 years (range, 1.4 to 15.5 years), the estimated 10-year overall survival and event-free survival are 94% (SE, 2.2%) and 88% (SE, 3.1%) respectively. There have been 12 treatment failures and six disease-related deaths. A very large mediastinal mass ( 50% of the maximal thoracic diameter) was associated with a 10-year event-free survival of 50% (SE, 14%) compared with 91% (SE, 4.0%) for smaller masses (P < .001). Late cardiopulmonary toxicity is largely absent, and the incidence of hypothyroidism is 14%. There have been no cases of secondary leukemia and four secondary solid malignancies observed to date.
MOPP/ABV and low-dose, extended-field radiotherapy is an effective treatment for pediatric Hodgkin's lymphoma. With median follow-up of 8.5 years, late cardiopulmonary effects and secondary malignancies from this treatment regimen are infrequent. Continued longitudinal observations, particularly for breast cancer in female patients and gonadotoxicity, will determine whether the goal of decreasing treatment-related complications while maintaining excellent survival has been achieved.
[Show abstract][Hide abstract] ABSTRACT: To determine the outcome in childhood renal cell carcinoma and the role of surgical and radiation treatment.
The records of 21 children with renal cell carcinoma were reviewed, 15 patients treated in the University of Toronto centers from 1959 through to 1997 and 6 patients treated in King Faisal Specialist Hospital, Riyadh, Kingdom of Saudi Arabia from 1975 through to 1998. The age was 3-17 (median 13) years. Systematic metastases were present at diagnosis in 5 patients. Regional nodal spread was present in 9 patients and 7 patients had localized disease alone. In the 16 M0 patients, the surgical treatment was radical nephrectomy (14 patients) partial nephrectomy (one patient) and wedge resection (one patient). Postoperative radiation treatment was utilized in 8 (50%) of these patients.
The 5 year survival rate for all patients was 52%, and for M0 patients was 70%. No patient with systematic metastases at diagnosis survived beyond 26 months. Four of 7 node negative patients and 8 of 9 node positive patients remained in first complete remission, with the duration of follow up 1-30 (Median 5) years. Seven of 8 M0 patients who did not receive adjuvant radiation therapy continued in first remission (3N0, 2NI, 2N2), compared with 5 of 8 patients who received postoperativeradiation treatment (1 N0, 2 NI, 2 N2).
The prognosis of localized renal cell carcinoma in childhood may be better than in the adult. Gross complete resection is required for long term survival. Elective postoperativeradiation treatment is not indicated.
Saudi medical journal 12/2004; 25(11):1583-6. · 0.59 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Gliomas of the optic pathways are rare childhood central nervous system tumors. The treatment approach is controversial because of its rarity and the slow and unpredictable growth rates of these lesions.
We reviewed 50 patients with the diagnosis of optic pathway low-grade gliomas treated between January 1980 and December 1995 at King Faisal Specialist Hospital and Research Center, Saudi Arabia. Thirty-five patients presented with chiasmatic/hypothalamic (posterior tumors), and 15 with optic nerve gliomas with or without chiasmal involvement (anterior tumors). Evidence of neurofibromatosis was present in 18 patients. Twenty-nine patients underwent surgery (total or partial resection), and 12 of these received postoperative radiotherapy (RT). Sixteen patients were treated with primary RT. The radiation dose varied between 42 and 54 Gy (median dose 50).
The overall actuarial survival rate was 87.5% at 5 years and 75% at 10 years, and the corresponding progression-free survival (PFS) rates were 69% and 62%. Patients with anterior tumors fared better than those with posterior tumors, with a 10-year PFS rate of 72% and 58%, respectively; the difference, however, was not statistically significant (p = 0.58). A PFS advantage was found in favor of patients with posterior tumors treated with RT (primary or postoperative) compared with no RT, with 5-year PFS rates of 68% vs. 42% (p = 0.03). This, however, did not translate into a survival advantage because of the success of salvage treatment.
In multivariate analysis, age (<3 vs. >3 years) emerged as the only significant determinant for PFS with patients <3 years old faring worse (p = 0.03). Neurologic and endocrine dysfunction are significant problems that need to be addressed.
International Journal of Radiation OncologyBiologyPhysics 08/2003; 56(3):807-12. DOI:10.1016/S0360-3016(02)04512-1 · 4.26 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: To evaluate prognostic factors for medulloblastoma.
One hundred and seventy-three consecutive patients with medulloblastoma, treated at King Faisal Specialist Hospital (KFSH) from 1988-1997, were reviewed. Eighty-four percent were children less than 15 years old. From 1988-1994, treatment was at the discretion of the investigator. From 1994-1998, patients entered a single-arm best practice protocol in which, in staged patients, the surgical intent was total resection, standard radiation treatment was defined, and adjuvant chemotherapy was given to a "high-risk" subset.
For 150 patients who completed surgical and radiation treatment, the 5-year survival rate was 58%, compared with 0% for 16 patients who were unable to start or complete radiation treatment. For staged patients, the 5-year survival was M0 + M1, 78% and M2 + M3, 21% (p < 0.0001). Other favorable significant prognostic factors were age >14 years and gross cystic/necrotic features in the primary tumor. The size of the primary tumor, the degree of hydrocephalus at diagnosis, the presence of residual tumor in the post-operative CT/MRI, and the functional status of the patient prior to radiation treatment were not significant factors.
Stage M0 + M1 was the most powerful favorable prognostic factor. In Saudi Arabia more patients present with advanced disseminated disease, 41% M2 + M3, than in the West, and this impacts adversely on overall survival. Total resection and standard radiation treatment were not sensitive prognostic factors in a treatment environment in which 78% of patients underwent at least 90% tumor resection and 60% received standard radiation treatment. In order to improve the proportion of patients able to complete radiation treatment, consideration should be given to limiting resection when the attainment of total resection is likely to be morbid, and to delaying rather than omitting radiation treatment in the patient severely compromised postoperatively.
International Journal of Radiation OncologyBiologyPhysics 07/2000; 47(3):573-84. DOI:10.1016/S0360-3016(00)00431-4 · 4.26 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: We wanted to evaluate survival and functional morbidity following radiation treatment of brain tumors in children less than 4 years old.
Outcome was evaluated for 222 children who were less than 4 years old when they were irradiated at University of Toronto Centres, 1958-1995. The status of the survivors with regard to focal neurological defects, vision, hearing, and education at last follow-up was recorded. In 23 adult survivors older than 21 years at last follow-up, information was obtained with regard to higher education, occupation, and living arrangements.
The overall 10-year survival rate was 40%, not significantly different than the 45% for 776 4-16-year-olds with irradiated brain tumors treated at the same institutions. Forty-five percent of the survivors had no major focal neurological, visual, or hearing defects. There were no major differences in the frequencies of these criteria or of schooling between 0-2- and 2-4-year-olds. Among adult survivors, older than 21 at last follow-up, 26% successfully completed higher education, 31% were in full-time employment, and 37% had never been employed. For medulloblastoma, the 5-year survival rate was 61% for 30 children less than 3 years old and treated from 1975-1995. This compared favorably with recent reports of survival following primary chemotherapy with delayed or omitted radiation treatment.
Radiation treatment of a young child with a brain tumor was associated with cure in 1 of every 3 patients. Unfortunately, quality of life for many survivors was not good. Only one of every 3 adult survivors was able to have a normal life-style. This shortfall was the result of focal neurological defects which were present from the time of first treatment, and of the long-term effects of radiation treatment.
The search for less toxic treatment remains appropriate, but is experimental and researchers must recognize that there may be a trade-off between morbidity and mortality.
Medical and Pediatric Oncology 01/1999; 31(6):506-11.
[Show abstract][Hide abstract] ABSTRACT: The treatment regimens at presentation and recurrence, the related morbidity, and survival rates were studied for children with craniopharyngioma who were treated at King Faisal Specialist Hospital & Research Center (KFSH&RC).
From 1975 to 1996, a total of 56 children younger than 18-years-old at diagnosis underwent treatment with surgery and/or radiotherapy for craniopharyngiomas at KFSH&RC. There was evidence that these patients had advanced disease at diagnosis; 36% of 51 patients whose visual status pretreatment was known were either blind or had major bilateral visual defects prior to treatment, and 36% of the 56 patients had diabetes insipidus. This report concentrates on 44 patients who had first definitive resection at KFSH&RC. Treatment policy was total resection if possible (17 patients), if not, subtotal resection (17 patients) or lesser procedures (10 patients). Five patients received postoperative irradiation after first definitive resection.
Ten-year-survival, and progression-free and event-free survival rates were 65%, 39%, and 29%, respectively. There were 9 postoperative deaths; 7 following the first procedure and 2 following resection for relapse, and 3 early deaths at home prior to relapse, following the first resection. None of the 22 patients who underwent less than total resection without postoperative radiation treatment were progression-free at 5 years, whereas the 5 patients who were irradiated remain progression-free.
A more selective approach to total resection, and the routine use of postoperative irradiation following lesser surgical procedures requires evaluation.
International Journal of Radiation OncologyBiologyPhysics 10/1998; 42(3):601-6. DOI:10.1016/S0360-3016(98)00257-0 · 4.26 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Following the introduction of CS-RT the survival rate for this malignant tumour rose from zero to approximately 50%. It appears that the major advances over the last twenty years associated with the introduction of CT/MRI, adequate staging, total resection, adjuvant therapy and improved radiation technique has only added 10-20% to the survival rate making it extremely difficult to evaluate the impact of these advances. While radiation treatment is currently omitted or delayed under the age of 36 months, due to enhanced neurocognitive toxicity, and replaced by maintained systemic therapy, the early encouraging results must stand the test of time in order to become standard practice. The treatment of a child with medulloblastoma with radiation treatment remains the corner stone of treatment while additional novel therapies are being developed.
Journal of Neuro-Oncology 08/1996; 29(1):45-54. DOI:10.1007/BF00165517 · 3.07 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: This study was undertaken to determine the treatment-specific incidence of second malignant tumours (SMT) in childhood Hodgkin's disease. The institutional databases at The Hospital for Sick Children, the Princess Margaret Hospital, and the Toronto-Bayview Regional Cancer Centre were reviewed for the years 1958-1993. Three hundred and forty-three consecutive newly diagnosed children were evaluated. The overall 30 year cumulative SMT incidence was 31%. The 20 year SMT incidence was greater for patients who relapsed (n = 129), 27%, compared with patients who remained relapse free (n=214), 13%. For patients with stage 1-3B disease who remained relapse free, the 10 year SMT rate was 7% for patients who were surgically staged and treated with extended field radiation treatment (EF RT) (35 G), compared with 3% in clinically staged patients treated with MOPP (six cycles) and EF RT (25-30 G). To date there is no significant difference in the oncogenicity of these treatment protocols. However, EF RT alone was less effective in disease control. For stages 1-3B, 62% of patients relapsed after EF RT alone compared with 18% after bimodal treatment. Therefore treatment intensification due to relapse was more frequent in the former group. The overall 10 year SMT incidence for patients treated with these protocols was 11% and 3%, respectively. The 20 year SMT incidence following EF RT alone was 24%. We conclude that SMTs were a common late complication in childhood Hodgkin's disease and are a limiting factor in the achievement of cure. The incidence of SMTs was increased in children who required retreatment and was minimal in children who remained in a first complete remission. Therefore the initial treatment strategy in childhood Hodgkin's disease must be to minimize the risk of relapse, in order to avoid the morbidity and mortality associated with both relapse and SMT induction, and to achieve this objective with a primary treatment protocol of low oncogenicity.
Medical and Pediatric Oncology 06/1996; 26(6):373-9. DOI:10.1002/(SICI)1096-911X(199606)26:6<373::AID-MPO1>3.0.CO;2-E
[Show abstract][Hide abstract] ABSTRACT: To determine the cause of death in children who survive more than 5 years after radiation treatment of a brain tumor.
Nine hundred and twelve consecutive children with a primary brain tumor irradiated at the Princess Margaret Hospital or Toronto-Bayview Regional Cancer Center from 1958 to 1991, were evaluated for long-term outcome.
Overall 10- and 20-year survival rates were 44% and 37%. Subsequent survival of 377 5-year survivors was, at an additional 10 and 20 years, 78% and 67%. Most (83%) deaths that occurred more than 5 years from diagnosis were a result of relapse of the original tumor. The 10-year survival rate subsequent to relapse was 9% when the first relapse occurred less than one year from diagnosis, 17% for 1-2 years, and 31% when the time to relapse was 3 years or greater. The cumulative actuarial incidence of, and death from, second malignant tumors at 30 years from diagnosis was 18% and 13%, respectively.
Death later than 5 years from diagnosis of a brain tumor in children is common and is usually due to progressive disease in slowly evolving low grade tumors. Death from a second malignant tumor becomes more frequent than death from the original tumor after 15 years from diagnosis.
International Journal of Radiation OncologyBiologyPhysics 03/1995; 31(3):445-51. DOI:10.1016/0360-3016(94)00393-Y · 4.26 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: This study was carried out to determine whether locoregional control of nasopharyngeal carcinoma in the young by irradiation has improved since 1975. Fifty-seven consecutive, previously untreated, patients, less than 30 years old, were diagnosed and treated at University of Toronto Hospitals between 1958 and 1990; 21 patients were treated before and 36 after 1975. Staging was as follows: M0, n = 54; M1, n = 3; T1 + T2, n = 26; T3 + T4, n = 31; N0, n = 10; N1 + N2a, n = 10; N2b + N2c, n = 24; N3, n = 13. All patients were irradiated. The primary tumour dose was 3500-7000 cGy (median 5450). Adjuvant chemotherapy was introduced in 1977. Subsequently, 10/26 (38%) M0 patients received this treatment. For all 57 patients, 10-year survival was 56%. For 52 M0 patients with complete follow-up data, 10-year survival was 63%; 10-year relapse free survival (RFS) was 61%. Age, sex, race, and histology were not significant variables. Ten-year RFS results were: T1 + T2, M0 (n = 24) 70%, T3 + T4, M0 (n = 27) 52%, (P = 0.25); N0-N2c (n = 41) 64%, N3 (n = 11) 54% (P = 0.31). Relapse occurred in 20/52 (38%) patients. Survival from the date of first relapse was 10% at 10 years. No patient with systemic relapse survived. Only 1/32 (3%) patients treated after 1975 developed an isolated locoregional relapse, giving a 10-year isolated locoregional relapse free rate (LR RFS) of 96%, compared with 5/20 (25%) prior to 1975, which gave a 10-year LR RFS of 75% (P = 0.05). Two of the five patients who relapsed before 1975 were salvaged by re-irradiation. Systemic relapse, either alone or combined with locoregional relapse, accounted for 6/11 (55%) relapses before 1975 and 8/9 (89%) after 1975. Ten-year systemic RFS was 90% for patients who received adjuvant chemotherapy (n = 10) and 72% for patients treated since 1977 by irradiation alone (n = 16) (P = 0.41). Isolated local relapse was exceptional in patients treated after 1975 (1/32).(ABSTRACT TRUNCATED AT 250 WORDS)
[Show abstract][Hide abstract] ABSTRACT: Optic pathway gliomas follow an unpredictable course. Some remain static for years; others increase rapidly in size and often lead to death. This unpredictability, along with the histological similarity of these tumors, has resulted in controversy about their management. We have reviewed the results of management of all 62 patients with a diagnosis of optic pathway/hypothalamic glioma treated at The Hospital for Sick Children during the years 1976–1990. Twelve patients received no direct treatment, and 3 only a biopsy. Six patients were treated with radiotherapy alone. Eight patients received radiotherapy following a biopsy. Seventeen patients were treated by resection alone and 16 had a resection followed by radiotherapy. Eight patients received chemotherapy in addition to other therapy, and in 5 of them the chemotherapy was given as an initial therapy. Forty-eight patients are well with their visual deficits but 7 of them are receiving hormone replacement therapy. Six patients have significant neurologic deficits and 8 have died.
[Show abstract][Hide abstract] ABSTRACT: Eighty-seven consecutive children with newly diagnosed optic glioma were managed at University of Toronto hospitals 1958-1990. Overall the 10-year survival, relapse-free survival and freedom from second relapse rates were 84%, 68% and 85%. Twenty-seven patients relapsed or progressed, of whom 40% were free of a second relapse 10 years after the first relapse. Fourteen patients had a second relapse. Thirteen are dead. None survived 5 years after second relapse. Patients with anteriorly located tumors (N = 35), which involved the optic nerve, or chiasm and optic nerves, fared better than those with posteriorly located tumors (N = 52) with spread beyond the chiasm, 10-year survival 95% versus 76%, (p = .02), 10-year relapse-free survival 80% versus 59% (p = .02), respectively. For posterior tumors primary irradiation was more effective than primary subtotal resection for prevention of subsequent relapse, 10-year relapse-free survival 75% versus 41% (p = .02), but salvage therapy was, in part, successful and multivariate analysis of prognostic factors influencing survival for posterior tumors indicated that neither primary resection nor primary irradiation were significant factors. For first relapse, primary irradiation and the presence of neurofibromatosis were the significant favorable factors. Since 1977 and for posterior optic glioma subtotal resection or surveillance were used in 21/29 (72%) patients compared with 4/23 (17%) previously. Ten-year survival rates before and after 1977 were 78% and 67% and 10-year relapse-free survival 64% and 56%, respectively.
International Journal of Radiation OncologyBiologyPhysics 02/1993; 25(2):215-25. DOI:10.1016/0360-3016(93)90342-S · 4.26 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Optic pathway gliomas follow an unpredictable course. Some remain static for years; others increase rapidly in size and often lead to death. This unpredictability, along with the histological similarity of these tumors, has resulted in controversy about their management. We have reviewed the results of management of all 62 patients with a diagnosis of optic pathway/hypothalamic glioma treated at The Hospital for Sick Children during the years 1976-1990. Twelve patients received no direct treatment, and 3 only a biopsy. Six patients were treated with radiotherapy alone. Eight patients received radiotherapy following a biopsy. Seventeen patients were treated by resection alone and 16 had a resection followed by radiotherapy. Eight patients received chemotherapy in addition to other therapy, and in 5 of them the chemotherapy was given as an initial therapy. Forty-eight patients are well with their visual deficits but 7 of them are receiving hormone replacement therapy. Six patients have significant neurologic deficits and 8 have died.
[Show abstract][Hide abstract] ABSTRACT: Five years ago, the use of preoperative chemotherapy for Wilms' tumor was adopted at this institution. Thirty children ranging in age from 5 months to 9 years had histologically confirmed Wilms' tumor (needle biopsy, n = 26; open biopsy, n = 4). Stage was determined by chest and abdominal computed tomography (CT) scan. Bilateral tumors were present in 6 children. All children received actinomycin D and vincristine from 3 weeks to 6 months before surgery. Seven children with bilateral tumors or stage IV disease also received adriamycin. CT-measured tumor masses shrunk in most cases. Subsequently, nephrectomy was performed in 23 patients and partial nephrectomy in 4, 2 of whom had bilateral disease. In 2 patients, residual bilateral well-differentiated epithelial tumor was not surgically resected. One child died before surgery. Reevaluation at delayed total or partial nephrectomy resulted in a downstaging of disease in 12 (41%). Further chemotherapy and radiation was based on the surgical stage. Postoperative chemotherapy (4 months to 2 years) was administered to all patients. The chest and/or abdomen were radiated in 12. Four patients (13%) died, 1 of radiation pneumonitis and 3 of the disease progression (2 with unfavorable histology, 1 of whom had bilateral disease). Two of 4 with unfavorable histology and 4 of 6 with stage IV disease have survived. It is concluded that this preoperative chemotherapy protocol is as effective as the National Wilms' Tumor Study (NWTS) protocol. The treated tumor is smaller, less friable, and easier to remove. Furthermore, because of downstaging, less radiation is necessary for cure.
Journal of Pediatric Surgery 09/1991; 26(8):949-53; discussion 953-6. DOI:10.1016/0022-3468(91)90842-H · 1.39 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: All patients with confirmed intracranial germ-cell tumors treated at the Hospital of Sick Children during the period January, 1952, to December, 1989, were reviewed. Of the 51 tumors reviewed, 16 were located in the suprasellar region, 32 in the pineal region, and three in both the pineal and the suprasellar regions. Forty-nine patients underwent surgical resection which was total in seven and partial in 20, and consisted of a biopsy in 22. Two patients were managed on the basis of serum and cerebrospinal fluid markers. Surgical tools such as the operating microscope, the ultrasonic surgical aspirator, and the laser beam allowed safe debulking and removal of the deep-seated tumors in the pineal region. There were no operative deaths in the 36 patients treated since 1972, who included 23 with pineal tumors. Twenty-five patients with germinomas received radiotherapy and had a 5-year survival rate of 85.1%. Thirteen patients with non-germinoma germ-cell tumors received radiotherapy and had a 5-year survival rate of 45.5%. On the basis of this review, the authors recommend resection of pineal and suprasellar germ-cell tumors in order to firmly establish an accurate histological diagnosis to guide the extent of adjuvant therapy. In the case of a pure germinoma without evidence of dissemination, adjuvant therapy consists only of local radiotherapy. On the other hand, for malignant non-germinoma germ-cell tumors, adjuvant therapy must include chemotherapy as well as craniospinal axis radiotherapy.
Journal of Neurosurgery 05/1991; 74(4):545-51. DOI:10.3171/jns.1991.74.4.0545 · 3.74 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Eighteen patients were diagnosed and treated for Stage IV-S neuroblastoma at The Hospital for Sick Children, Toronto between January 1971 and December 1988. All patients were 6 months of age or younger at diagnosis. Nine patients (50%) have remained disease free with a mean follow-up of 9.3 years. Of the seven patients under 6 weeks of age at presentation, four presented in the early neonatal period and died, three due to mechanical complications related to progressive disease, and one due to late recurrence. The remaining three patients under 6 weeks of age, two of whom had skin involvement at diagnosis, are alive and disease free. Six of the 11 patients over 6 weeks of age at presentation survived, combined modality therapy (CMT) being more effective than single modality treatment. N-myc was studied from tumor tissue at diagnosis in four patients and was amplified in three (25x, 25x, 100x), all of whom had late disease progression and died. The patient with a single gene copy has no evidence of disease 24 months following diagnosis. Our study confirms the heterogeneity described in this clinically defined group of patients. Because of it, management of Stage IV-S neuroblastoma cannot be uniform and until further development of a subclassification, or a reclassification based on molecular biologic markers is developed, pediatric oncologists will regularly be confronted with a decision whether or not to treat a newly presenting patient that fits into the clinical classification IV-S.
Medical and Pediatric Oncology 02/1991; 19(6):467-72.