Showing papers in "Pediatric Hematology and Oncology in 1998"
TL;DR: By the age of 2 years the infants of this study had not reached adult serum AFP levels (0-6 ng/ml), and reference values and factors associated with serum AFP elevation in infants were established.
Abstract: The aim of this study was to establish reference values and factors associated with serum AFP elevation in infants. Five hundred twenty-four samples collected from infants up to the age of 2 years at the University Hospital Dusseldorf (Germany) were analyzed. At birth mean serum AFP levels were 41,687 ng/ml in 256 term babies and 158,125 ng/ml in 90 premature babies born before the 37th gestational week, excluding samples from children with factors known to be associated with AFP elevation. In the first 4 weeks of life, AFP levels decreased by 50% in 5.1 days in term babies. Between day 180 and 720 of life, AFP levels up to 87 ng/ml were within the 95.5% interval (assumed logarithmic normal distribution) with a mean of 8 ng/ml without a further decline. By the age of 2 years the infants of this study had not reached adult serum AFP levels (0-6 ng/ml).
198 citations
TL;DR: Multimodal intervention may be required to manage fastidious hemangioendotheliomas of childhood, achieve clinical improvement, and prevent further morbidity.
Abstract: We present the management challenge provided by a patient with kaposiform hemangioendothelioma associated with Kasabach-Merritt phenomenon A female child presented at 14 months of age with an ecchymotic swelling of her right upper arm and axilla Subsequently, she developed profound thrombocytopenia and hypofibrinogenemia (Kasabach-Merritt phenomenon) Biopsy of the lesion revealed kaposiform hemangioendothelioma, which has been reported as the predominant pathologic diagnosis associated with Kasabach-Merritt phenomenon To achieve involution of the lesion and preserve function of the arm, the following interventions were involved: embolization, systemic interferon, cyclophosphamide, epsilon aminocaproic acid, and compression therapy The clinical management of this patient was formidable until we arrived at the proper combination of therapies Multimodal intervention may be required to manage fastidious hemangioendotheliomas of childhood, achieve clinical improvement, and prevent further morbidity
71 citations
TL;DR: Although most of the children in the ALL group were well adapted, one third of them had low CBCL Social Competence scores relative to nonclinical norms, and low speed and hypoactivity seemed to limit the majority of these children in school and daily life activities.
Abstract: The cognitive and psychosocial functions of 16 children with brain tumor, treated with wholebrain or local radiotherapy, were studied. Fifteen nonirradiated children with acute lymphatic leukemia (ALL) served as a comparison group. Mean Wechsler Intelligence Scale for Children--Revised Full Scale IQ score and mean Visual-Motor Integration Test (Beery VMI) standard score were significantly decreased in the brain tumor group. The Achenbach checklists were filled in by mothers (Child Behavior Checklist, CBCL) and teachers (Teacher's Report Form). Overall adjustment was rated lower in the children with brain tumor. Although most of the children in the ALL group were well adapted, one third of them had low CBCL Social Competence scores relative to nonclinical norms. On a visual analog scale a significantly decreased tempo was assessed by mothers and teachers in the children with brain tumor, and low speed and hypoactivity seemed to limit the majority of these children in school and daily life activities.
48 citations
TL;DR: The salient clinical and epidemiological characteristics of retinoblastoma (RB) in India is examined, thereby highlighting the problems encountered there and a possibility of difference in the biology of the tumor seen in these patients.
Abstract: This study examined the salient clinical and epidemiological characteristics of retinoblastoma (RB) in India, thereby highlighting the problems encountered there. The epidemiological characteristics of 296 patients with RB over 8 years were evaluated using hospital records and postal follow-ups. Unilateral disease was seen in 61.8% of patients. The overall median age at presentation was 3.5 years (3.5 years for unilateral RB and 1.0 years for bilateral RB). The male/female ratio was 1.4:1. The median duration of symptomatic disease was 8 months. Consanguineous marriage was seen in 17% and family history of RB was noted in 1.7% cases. Also, 2% had a history of other malignancy in the family. Associated congenital malformation was seen in 10.5% of cases. A second malignancy was seen in 0.67% of cases at a mean duration of 4.5 years after completion of therapy. A predominance of advanced-stage disease (74.5% had Reese-Ellsworth group IV and V disease) was seen in our series. Only 43.6% of patients had disease localized to the globe without any infiltration/invasion. The majority of cases had advanced-stage disease at presentation and came from the underprivileged class of society. Patients with bilateral RB presented much earlier than those with unilateral disease. In patients with unilateral RB, higher age at presentation as well as advanced disease may be related to much delay in seeking medical attention. In view of the advanced stage at presentation, there also exist a possibility of difference in the biology of the tumor seen in these patients.
47 citations
TL;DR: Close clinical monitoring of children for early diagnosis of this complication is recommended and Ultrasonography can be used as a corroborative investigation for diagnosis along with serum chemistries.
Abstract: A major complication of L-asparaginase therapy in acute lymphoblastic leukemia (ALL) is pancreatitis. Among the 915 cases of ALL seen over last 10 years, 19 (2%) developed pancreatitis. Median age was 5.5 years with a median duration of symptoms of 4 weeks. All cases had raised serum amylase and lipase. Pancreatitis was not related to the dose/duration of L-asparaginase therapy. Ultrasonography can be used as a corroborative investigation for diagnosis along with serum chemistries. Close clinical monitoring of children for early diagnosis of this complication is recommended.
45 citations
TL;DR: It is concluded that on the basis of clinical features, two distinct subgroups could be identified in terms of SI: patients with clinically relevant severe SI had a better prognosis, a higher DI, and more frequently a common ALL than patients without SI.
Abstract: Skeletal radiographic abnormalities are common in children with acute lymphoblastic leukemia (ALL) The impact of severe skeletal involvement (SI) on survival and the correlation between SI and biological markers were analyzed Therefore, radiographs and medical charts of 106 ALL patients who received a skeletal survey at the time of diagnosis and were treated at the University Children's Hospital Wurzburg between 1974 and 1995 were reviewed On the basis of the skeletal survey, SI was quantified using a score Fifty-nine patients (55%) showed radiographic abnormalities defined as metaphyseal banding (48%), periosteal reactions (11%), osteolysis (33%), osteosclerosis (31%), or osteopenia (22%) Children with severe SI (n = 32) presented with a higher rate of severe radiographic abnormalities such as geographic destructive osteolysis (37%; P 116 No patient with a DI < 10 presented with severe SI The number of radiographic abnormalities in patients with SI correlated with the DI (rho: 046; P < 001) However, patients with euploidy (DI = I) and severe SI also had a higher (P = 05) survival rate (70 +/- 18%; n = 15) than euploid patients without SI (49 +/- 11%; n = 24) Of the patients with severe SI, 78% had common ALL and 22% had an ALL type other than common ALL (P < 05) In patients with ALL types different from common ALL, severe SI was also associated with higher survival rates We conclude that on the basis of clinical features, two distinct subgroups could be identified in terms of SI Patients with clinically relevant severe SI had a better prognosis, a higher DI, and more frequently a common ALL than patients without SI However, the impact of severe SI on prognosis was independent of DI and type of leukemia
38 citations
TL;DR: The poor prognosis in adrenocortical carcinoma in childhood is confirmed, but a complete resection is compatible with cure of the primary disease.
Abstract: In a 41-year period, 18 children with a diagnosis of an adrenal cortical tumor were identified (14 carcinoma : 4 adenoma). The majority of patients had clinical signs of endocrine dysfunction at presentation, with virilization (11 patients) and a cushingoid appearance (8 patients) the commonest findings. Abnormal biochemical activity was identified in 16 tumors (94%). The primary treatment in 17 patients was surgical. In addition, 12 children, all with carcinomas, had radiotherapy. Of those children with a carcinoma, 12 are dead, with a median survival of 52 months (range 1–317 months). The three second primary tumors all developed at sites within the field of previous radiotherapy, and proved fatal at 127, 176, and 317 months (median 207 months). This series confirms the poor prognosis in adrenocortical carcinoma in childhood, but a complete resection is compatible with cure of the primary disease. The frequency of second, fatal, primary tumors is of particular concern and long-term follow-up is mandator...
38 citations
TL;DR: Infections during the chemotherapy of ALL were a significant cause of morbidity in children, but mortality was low.
Abstract: In a retrospective analysis we evaluated the occurrence of infections in 59 children with acute lymphoblastic leukemia (ALL) during the entire duration of their anticancer chemotherapy. We recorded a total of 245 infection episodes, 118 (50%) being during neutropenia and 119 (50%) during nonneutropenia. The infections most commonly detected during neutropenia were fevers of undetermined origin (36%), clinically or microbiologically defined focal infections (33%), and bacteremias (28%). During nonneutropenia, upper respiratory tract infections (55%) were the most common. Patients needed hospitalization for infections for a total of 1951 days (i.e., a mean of 33 days per patient) and the mean number of infection episodes was 4.2 per patient. Recurrent fever developed in 21% of the children with bacteremia. Mortality caused by bacteremias was 10%. Infections during the chemotherapy of ALL were a significant cause of morbidity in children, but mortality was low.
34 citations
TL;DR: Factors were determined that influence the occurrence and clinical course of AON in ALL patients that could help in developing therapeutic strategies for the prevention of progressive AON.
Abstract: Joint destruction caused by aseptic osteonecroses (AONs) is a severe complication in acute lymphoblastic leukemia (ALL). Here, factors were determined that influence the occurrence and clinical course of AON in ALL patients. Clinical data of 121 patients were correlated with the occurrence of AON. Magnetic resonance imaging was performed in patients with bone pain. Areas of AONs were correlated with the clinical course of AON. Ten patients presented with clinical symptoms of AON and 9 of 10 patients were graded as high-risk ALL compared with 46 of 111 patients without AON (X2, P <. 05). In 10 ALL patients 66 AONs were identified by MRI. In some patients up to 14 different AON sites were observed. The courses of AONs varied in individual patients, suggesting that necrosis-specific factors might be responsible. A size above 900 mm2 was significantly associated with progressive AON (P <. 01). The more intensive treatment regimen in high-risk ALL patients might contribute to the development of AON. The clinic...
32 citations
TL;DR: It is suggested that the prescription of vancomycin at 40 mg/kg daily, divided into four daily doses, is safe and will provide a peak blood level of van comycin sufficient to cover the broad spectrum of staphylococcal bacteria.
Abstract: Thirty children suffering from different types of malignancies, neutropenic fever, and suspected staphylococcal bacteremia were evaluated for the pharmacokinetics of vancomycin in steady-state conditions and compared with eight children suffering from proven methicillin-resistant staphylococcal infection. All the studied population received intravenous vancomycin at 40 mg/kg daily divided into four daily doses. The individual pharmacokinetic parameters were calculated using a one-compartment model for two blood vancomycin samples. The mean (+/- SD) half-time (t1/2, hours), clearance (L/h/kg), Vss (L/kg), Cmax (microgram/mL), and Cmin (microgram/mL) were 10.5 (7.9) and 14.9 (9.1) hours; 0.11 (0.14) and 0.06 (0.06) L/h/kg; 0.62 (0.33) and 1.3 (0.6) L/kg; 28.3 (11.8) and 22.3 (9.8) micrograms/mL; and 5.7 (6.0) and 7.4 (4.8) micrograms/mL for the malignancy and control groups, respectively. The malignancy group had a significantly shorter t1/2 (P = .005), higher clearance (P = .005), and lower Cmin (P = .03) in comparison with the control group. It is suggested that the prescription of vancomycin at 40 mg/kg daily, divided into four daily doses, is safe and will provide a peak blood level of vancomycin sufficient to cover the broad spectrum of staphylococcal bacteria. The vancomycin dose should be individualized, based on an individual pharmacokinetic profile.
29 citations
TL;DR: It is concluded that P-gp-mediated multiple drug resistance is not a factor in a slow response to ALL induction therapy, and the bone marrow aspirate slides for early response to treatment were analyzed.
Abstract: In childhood acute lymphoblastic leukemia (ALL), early response to treatment is an important prognostic factor and drug resistance is a major cause of poor outcome. One of the most investigated resistance mechanisms is P-glycoprotein (P-gp)-mediated multiple drug resistance (MDR). We analyzed P-gp using flow cytometry with monoclonal antibody JSB1 in a series of 118 children with ALL, 103 at diagnosis and 15 at relapse. Increased P-gp expression was found in 55 (53%) patients at diagnosis and in 11 (73%) at relapse. We also analyzed the bone marrow aspirate slides for early response to treatment in a central review. No correlation was found between P-gp and early response. Patients with T-ALL had higher P-gp levels than the others, 5.3% versus 1.0% (P -.002). We conclude that P-gp-mediated multiple drug resistance is not a factor in a slow response to ALL induction therapy.
TL;DR: In the present study, protein expressions of MDM2 and p53 were investigated by immunohistochemistry from bone marrow samples in 23 patients with acute lymphoblastic leukemia aged 1-13 years at diagnosis, with p53 protein overexpression detected in only one case, while overexpressing MDM 2 was detected in samples from five patients.
Abstract: Mutation of the TP53 gene is one of the most common molecular alterations in a variety of tumors, but it occurs infrequently in childhood and adult hematological malignancies. Protein accumulation often results from mutations that lead to inactivation of the p53 protein. Other causes of functional inactivation of the p53 protein include stabilization of p53 via proteins such as MDM2, an oncoprotein capable of forming specific complexes with p53. In the present study, protein expressions of MDM2 and p53 were investigated by immunohistochemistry from bone marrow samples in 23 patients with acute lymphoblastic leukemia aged 1-13 years at diagnosis. p53 protein overexpression was detected in only one case, while overexpression of MDM2 was detected in samples from five patients. All five patients overexpressing MDM2 belonged to a group with unfavorable prognostic signs at diagnosis and three of them relapsed or died within 6 months after diagnosis.
TL;DR: In the iron-deficiency group the production of IL-2 was found to be significantly lower than that in controls and became normal after iron supplementation (P < .001), but there was no difference in serum levels ofIL-6 in iron deficiency anemia before and afterIron supplementation.
Abstract: The aim of this study was to investigate the serum levels of interleukin-2 (IL-2) and interleu-kin-6 (IL-6) in children with iron deficiency anemia before and after iron supplementation. Twenty-five children with iron deficiency anemia 6 months to 3 years of age were included in the study. Ten age- and sex-matched healthy children constituted the control group. In the iron-deficiency group the production of IL-2 was found to be significantly lower than that in controls and became normal after iron supplementation (P . 05).
TL;DR: Significantly prolonged disease-free survival was seen in patients who received ATRA with chemotherapy, and ATRA was well-tolerated by these patients.
Abstract: Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloblastic leukemia (AML). In this report, we present the clinical features, management, and outcome of pediatric patients with APL treated with all-trans-retinoic acid (ATRA). Of 52 newly diagnosed cases of APL between February 1992 and December 1996, 15 were in the pediatric age group (younger than 15 years). Four patients were treated with ATRA alone and 11 were allocated to receive ATRA followed by chemotherapy. Eighty-six percent of the patients achieved a complete response. The patients who received ATRA alone as maintenance therapy had relapses with a median duration of remission of 8 months (range 6-12). The patients who received ATRA, followed by consolidation chemotherapy, had a prolonged duration of remission, with a median of 20 months (range 13-28). In addition, rapid correction of coagulopathy was observed in these patients. The median duration for correction of coagulopathy was 7 days (range 5-11) and the median duration for recovery from neutropenia after chemotherapy was 10 days (range 7-20). Two major side effects of ATRA were hyperleukocytosis and retinoic acid syndrome. Significantly prolonged disease-free survival was seen in patients who received ATRA with chemotherapy. APL is not uncommon in the pediatric age group. ATRA was well-tolerated by these patients. Consolidation with chemotherapy helps in prolonging the disease-free survival in patients with APL in comparison to treatment with ATRA alone.
TL;DR: A 10-year-old boy with hyperimmunoglobulin E (HIE) syndrome was admitted to the hospital due to intermittent fever and a growing neck mass noted for 3 months, and pathological examination of the lymph node disclosed Hodgkin's disease (nodular sclerosis).
Abstract: A 10-year-old boy with hyperimmunoglobulin E (HIE) syndrome was admitted to the hospital due to intermittent fever and a growing neck mass noted for 3 months. He had had chronic eczema and recurrent skin infections since infancy. At age 8, the diagnosis of HIE was established when a pneumatocele was found in the presence of extremely elevated serum IgE levels (7842 IU/mL). He also had defective T-lymphocyte function, manifested by cutaneus anergy, as well as abnormal proliferative response to mitogenic stimuli. Chemotactic function of neutrophils was normal. Pathological examination of the lymph node disclosed Hodgkin 's disease (nodular sclerosis). A high index of suspicion for lymphoma should be given in patients with HIE syndrome who present with lymph node enlargement.
TL;DR: A case of a 20-year-old male with Gorlin's syndrome, developing multiple basal cell carcinomas within irradiation fields 10 years after the treatment of medulloblastoma is reported.
Abstract: We report a case of a 20-year-old male with Gorlin's syndrome (nevoid basal cell carcinoma syndrome), developing multiple basal cell carcinomas within irradiation fields 10 years after the treatment of medulloblastoma.
TL;DR: The authors retrospectively analyzed 863 episodes of neutropenia and fever in 635 children with cancer or hematological disease hospitalized between October 1988 and November 1994 and revealed that the presence of a severe noninfectious complication, severe neutropania, and positive blood culture correlated with high mortality rate.
Abstract: The authors retrospectively analyzed 863 episodes of neutropenia and fever in 635 children with cancer or hematological disease hospitalized between October 1988 and November 1994. The most frequent underlying diseases were solid tumors (45%) and acute lymphoblastic leukemia (29%). Clinical site of infection could be determined in 454 (53 %) episodes. Bacteremia was documented in 114 (13%) cases. Gram-positive cocci were the microorganisms most frequently isolated (47% of the cases). Noninfectious complications could be determined in 140 (16%) episodes, and were mainly severe bleeding and metabolic impairment. The episodes were divided in two groups for comparative evaluation: group A, 404 episodes, study period 1988-1991, and group B, 459 episodes, 1992-1994. According to the results, more patients in group A than group B were younger than 1 year old and had profound neutropenia; fewer patients in group A than group B had an endovascular catheter, a higher frequency of manifest clinical site of infection...
TL;DR: Vitamin B12 deficiency should be considered in the differential diagnosis of a child presenting with hyperpigmentation and macrocytic red cell indices.
Abstract: The authors report on 2 children with pernicious anemia, sisters, who presented with hyper-melanosis as one of the clinical manifestations. The hypermelanosis disappeared with adequate treatment of vitamin B12 deficiency. Vitamin Big deficiency should be considered in the differential diagnosis of a child presenting with hyperpigmentation and macrocytic red cell indices.
TL;DR: In children and adolescents increased APA titers may occur in a variety of diseases, including autoimmune or autoimmune-like diseases or infections.
Abstract: The prevalence of antiphospholipid antibodies (APAs) has not yet been studied in children and adolescents with various diseases. We assayed plasma samples of 203 children and adolescents, aged 0.1 to 21 years (median 6 years), by enzyme-linked immunosorbent assay detecting immunoglobulin G (IgG) and IgM antibodies directed against cardiolipin, phosphatidylserine, and phosphatidic acid. According to their diagnosis children were divided into five groups: 10 patients with autoimmune and autoimmune-like diseases (group 1); 88 patients with infections (group II); 20 patients with metabolic diseases (group III); 65 children with various other diseases (group IV); 20 healthy children without physical illness (group V). Results were compared for statistical significance using Fisher's exact test. APAs were found in 65 of 203 patients. In all groups of patients the prevalence of APAs was increased compared with healthy children. The highest prevalence of APAs was found in patients with, autoimmune or autoimmune-l...
TL;DR: Results suggest that self-report rating scales can be useful assessment instruments if used as complementary tools and case examination of depressed patients suggests that illness and treatment factors may not be primary risk factors for the development of psychological morbidity.
Abstract: Adolescents with cancer need to cope with their disease and treatment while accomplishing the tasks unique to this developmental period. In this pilot study, the prevalence of psychological morbidity among adolescents with cancer was examined. In addition, assessment methods and risk factors were explored. Forty-three recently diagnosed adolescents with cancer completed the Hospital Anxiety and Depression Scale (HADS), the Beck Depression Inventory (BDI), and the Rotterdam Symptom Checklist (RSCL) and underwent a full psychiatric assessment. In the psychiatric interviews, 9% of the sample was diagnosed with a depressive mood disorder. Results suggest that self-report rating scales can be useful assessment instruments if used as complementary tools. Psychosocial assessment of the adolescent with cancer is nevertheless difficult. This appears to be due to an atypical symptomatology pattern and a tendency toward the masking of distress. Case examination of depressed patients suggests that illness and treatme...
TL;DR: The results indicate that patients with AT are at risk for developing multiple primary cancers, including those of the parotid and thyroid gland, and should be evaluated for such primaries.
Abstract: This study describes the clinicopathologic features of parotid and thyroid gland cancers in patients with ataxia-telangiectasia (AT). The medical records of 412 AT patients were reviewed to identify those patients who developed parotid or thyroid gland cancers. Presenting features, diagnoses, types of therapy, risk factors, and other primary cancers were analyzed. Five patients with parotid or thyroid gland cancers were identified. Three had parotid (2 mucoepidermoid and 1 acinic cell) and 2 had thyroid gland (1 papillary and 1 follicular) cancers. Four patients presented with head and neck masses and 1 had an occult papillary thyroid carcinoma. Four patients had more than one primary cancer. The only mode of therapy was surgery. The 2 patients with mucoepidermoid carcinoma had complete parotidectomies. One is alive without any evidence of disease 12 months after diagnosis and 1 died of refractory lymphoma without any evidence of mucoepidermoid carcinoma at autopsy. The patient with acinic cell carcinoma ...
TL;DR: Adoptive immunotherapy with donor leukocyte transfusion with appropriate numbers of CD3-positive T cells was effective for the EB-LPD, achieving almost complete recovery 1 year later without any findings of graft-versus-host disease.
Abstract: We report a case of a 16-month-old Wiskott-Aldrich syndrome (WAS) patient with a WASP gene mutation who received human leukocyte antigen (HLA)-matched, unrelated allogeneic bone marrow transplantation (BMT) followed by an Epstein-Barr virus-associated lymphoproliferative disorder (EB-LPD), diagnosed by clinical findings, polymerase chain reaction detection of the EB virus genome, and spontaneous lymphocyte proliferation of donor cell origin. EB-LPD is one of frequent lethal complications in HLA-mismatched or unrelated BMT in this syndrome. Adoptive immunotherapy with donor leukocyte transfusion, including appropriate numbers of CD3-positive T cells, was effective for the EB-LPD, achieving almost complete recovery 1 year later without any findings of graft-versus-host disease.
TL;DR: Although the expression of Bcl-2 varied widely from 7 to 80 × 103 MESF units, no significant difference was found, in the mean value, between the patients with acute lymphoblastic leukemia and those with acute myeloblasticukemia.
Abstract: Bcl-2 expression and its prognostic value were evaluated in 42 children with acute leukemia. The Bcl-2 expression of the leukemic blast cells was measured quantitatively by flow cytometry and was further analyzed by the simultaneous immunostaining of Bcl-2 with the surface membrane antigens, DNA, Ki-67 antigen. All of the cases showed a consistent expression of Bcl-2 protein; virtually all leukemic lymphoblasts were Bcl-2 positive. Although the expression of Bcl-2 varied widely from 7 to 80 × 103 MESF units, no significant difference was found, in the mean value between the patients with acute lymphoblastic leukemia and those with acute myeloblastic leukemia. In more than half of the patients with AML, intraclonal heterogeneity of Bcl-2 expression was observed. The expression of Bcl-2 showed no apparent fluctuations during the different phases of the cell cycle. However, the proportion of Bcl-2-positive and -negative cells during the cell cycle was different between ALL and AML patients. In the ALL patien...
TL;DR: It is concluded that vincristine and actinomycin D were the cause of this rare from of hepatotoxicity and that chemotherapy for the underlying malignant disease could be given safely after clinical recovery.
Abstract: We report two pediatric cases who developed veno-occlusive disease—like hepatotoxicity while receiving chemotherapy for Wilms' tumor and clear cell sarcoma of kidney. The chemotherapeutics, including vincristine, actinomycin D, and epirubicin in case 1 and vincristine and actinomycin D in case 2, were given before the hepatotoxicity developed. Other possible causes of hepatotoxicity were excluded. Recovery was observed with supportive therapy after 2 and 1 weeks, respectively. After recovery, the children tolerated continued chemotherapy without any decrease in the doses of drugs. We conclude that vincristine and actinomycin D were the cause of this rare form of hepatotoxicity and that chemotherapy for the underlying malignant disease could be given safely after clinical recovery.
TL;DR: The results suggest that the ongoing nutritional status of children with malignancy undergoing chemotherapy is best assessed using simple clinical parameters.
Abstract: The nutritional status of a child on cancer therapy influences both tolerance of and response to treatment. However, it is difficult to assess nutritional status on a daily basis because an accurate quantitation of the calorie intake is difficult. Anthropometric and biochemical parameters are prone to error and often reflect past rather than current nutritional status. In practice, a subjective clinical assessment is usually relied upon. This study objectively appraises the value of such an assessment. Based on clinical symptoms that alter oral intake and absorption of food, a scoring system was designed to assess nutritional status on a day to day basis. A symptom score (SS) of 10 implied "normality"; 0 indicated maximum debility. Over a 2-year period 511 daily scores were recorded in 30 patients aged 0.7-17.5 years. Patients were studied at presentation and during treatment for acute lymphoblastic leukemia (ALL, n = 14; solid tumors receiving megatherapy with autologous bone marrow rescue (ABMR, n = 8), and chemotherapy for different tumors (miscellaneous, n = 8). The SS was compared with other nutritional parameters, including sequential anthropometric indices, serum albumin, insulin-like growth factor-I (IGF-I), IGF binding protein-3 (IGFBP-3), and whole-body protein turnover (WBPT) using [1-(13) C]leucine. The mean SS was reduced at diagnosis for all leukemic patients (median score = 8), improved during first remission (p < 0.002), fell to a minimum during febrile neutropenia (p = 0.0009), and improved with clinical and hematological recovery (p = 0.0009). A reduction in SS was related to fever (p < 0.001) and a fall in neutrophil count (p < 0.001). There was no correlation with anthropometric indices or IGF-I and IGFBP-3 levels. Paired WBPT studies in 9 patients showed that SS correlated well with protein breakdown (p = 0.026). The results suggest that the ongoing nutritional status of children with malignancy undergoing chemotherapy is best assessed using simple clinical parameters.
TL;DR: In this paper, the authors identified the beta s-globin gene haplotypes of 85 patients with sickle cell disease attending the Dubai Thalassemia Center and assessed the influence of haplotype, alpha-thalassemia, and fetal hemoglobin on the clinical presentation.
Abstract: We have identified the beta s-globin gene haplotypes of 85 patients with sickle cell disease attending the Dubai Thalassemia Center and assessed the influence of haplotype, alpha-thalassemia, and fetal hemoglobin on the clinical presentation. Identification of the beta s haplotypes was based on mutation analyses in the promoter sequences of the G gamma- and A gamma-globin genes. The Arabian-Indian haplotype was found in 52% of the beta s chromosomes, whereas the remaining were the Bantu and Benin haplotypes. Those with the Arabian-Indian haplotype in this group had a significantly higher fetal hemoglobin (Hb F) level (mean 27%) and a milder clinical course. In contrast, those with the African haplotypes, Bantu and Benin, expressed relatively lower Hb F levels (mean 11.3%), with a severe clinical presentation. Coinheritance of alpha-thalassemia trait in the African haplotypes had an ameliorating effect on hemolytic episodes, but vaso-occlusive crises were more frequent.
TL;DR: A 14-year-old girl with an unusual extramedullary presentation of acute nonlymphocytic leukemia is described, who presented with involvement of both lacrimal glands, parotid and submandibular salivary glands, and both breasts.
Abstract: A 14-year-old girl with an unusual extramedullary presentation of acute nonlymphocytic leukemia is described. She presented with involvement of both lacrimal glands, parotid and submandibular salivary glands, and both breasts. Biopsy of the submandibular salivary gland, breasts, and bone marrow was suggestive of acute monocytic leukemia. She was alive for 5 months with supportive treatment.
TL;DR: Examination of the incidence of paraganglioma in the childhood population of the West Midlands region of the United Kingdom since 1957 found 4 cases and histopathological examination suggested the benign nature of the tumor, implying that surgical excision was sufficient treatment.
Abstract: Paraganglioma (extra-adrenal pheochromocytoma) is a rare tumor, particularly in childhood. Those in the female genital tract are exceptionally rare, with only 9 cases reported in detail since 1926. All were seen in adults and only two arose in the vagina. This study examined the incidence of this tumor in the childhood population of the West Midlands region of the United Kingdom since 1957 and found 4 cases: 2 abdominal, 1 para-aortic, and 1 carotid body. A recent additional case is reported in the vagina of a child aged 11 years, who presented with heavy vaginal bleeding in the absence of hypertension. Initial diagnosis suggested rhabdomyosarcoma and near complete excision was carried out. Since this is the first such case to be described in a child, the outcome can only be assumed. Although histopathohgical examination suggested the benign nature of the tumor, implying that surgical excision was sufficient treatment, close follow-up is recommended.
TL;DR: Two cases of multifocal desmoid tumors arising in two teenagers with different, separate lesions developed in the same limb over a period of several years, one of which recurred on several occasions.
Abstract: We present two cases of multifocal desmoid tumors arising in two teenagers. Different, separate lesions developed in the same limb over a period of several years, one of which recurred on several occasions. The literature regarding the therapeutic aspects of multifocal fibromatosis is reviewed and discussed.
TL;DR: It is suggested that PVB chemotherapy after orchiectomy is an affective and safe regimen for stage I yolk sac tumor of testis in children younger than 2 years and two or three courses of PVB could be enough.
Abstract: Yolk sac tumor is the most frequent germ cell tumor of testis in children. For stage I yolk sac tumor of testis in children younger than 2 years, high inguinal orchiectomy alone has been the standard treatment, with a cure rate of at least 75%. Here, we compare the treatment results of receiving chemotherapy or no chemotherapy after orchiectomy, to analyze the role of chemotherapy. From February 1987 to January 1997, 22 children younger than 2 years, with stage I yolk sac tumor of testis, were included in the study. All patients had high inguinal orchiectomy without retroperitoneal lymphadenectomy. Initial diagnostic imaging studies included computed tomographic scan of abdomen, chest radiography, and long bone survey. Clinical stage I was defined as a tumor completely resected with no evidence of local regional lymph node involvement or distant metastases. Serum alpha-fetoprotein (AFP) was assessed at diagnosis. After orchiectomy, diagnosis, and staging, patients were stratified into two treatment groups, with or without chemotherapy, according to the decision of the parents. Ten children received chemotherapy consisting of cisplatin, vinblastine, and bleomycin (PVB, modified "Einhorn regimen") for 12 weeks. The remaining 12 patients were followed up according to a "wait and see" policy. Determination of AFP was performed monthly during the first postoperative year, every other month during the second year, every 3 months during the third year, every 6 months during the fourth year, and yearly until the fifth postoperative year at least. The duration of follow-up ranged from 3 months to 119 months (median, 53 months). The Kaplan-Meier plot estimated an overall survival rate of 91.6% at 7 years after diagnosis. Among the 12 patients without chemotherapy, 2 children had relapses at 4 and 6 months after diagnosis, respectively. One was cured with PVB chemotherapy. The other patient died with refractory lung metastasis, in spite of intensive multimodality salvage therapy. The Kaplan-Meier plot showed a survival rate of 80% at 7 years and a relapse-free survival rate of 81.8% at 5 years after diagnosis. All children receiving chemotherapy were alive and free from relapse. There was no significant treatment-related toxicity. Our results may suggest that PVB chemotherapy after orchiectomy is an affective and safe regimen for stage I yolk sac tumor of testis in children younger than 2 years. Instead of four courses of PVB as used here, two or three courses could be enough. To elucidate the necessity for chemotherapy and to determine the number of courses of PVB needed (if chemotherapy is given), a randomized study of more cases is warranted.