17 results on '"Qaddoumi I"'
Search Results
2. Pediatric Neurosurgical Capacity for the Care of Children With CNS Tumors Worldwide: A Cross-Sectional Assessment.
- Author
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Roach JT, Qaddoumi I, Baticulon RE, Figaji A, Campos DA, Arredondo L, Boop FA, and Moreira DC
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- Humans, Child, Cross-Sectional Studies, Developing Countries, Neurosurgery, Central Nervous System Neoplasms diagnosis, Central Nervous System Neoplasms surgery, Glioma
- Abstract
Purpose: Efforts to address inequities in the treatment of pediatric CNS tumors and the burden of childhood cancer globally have prompted the designation of low-grade glioma as one of six index cancers for the World Health Organization Global Initiative for Childhood Cancer. Understanding the importance of neurosurgical interventions and evaluating pediatric neurosurgical capacity may identify critical interventions to improve outcomes for children with low-grade glioma and other CNS tumors., Methods: An online, cross-sectional survey assessing pediatric neurosurgical practice and capacity was distributed to members of the International Society of Pediatric Neurosurgery. The survey included 36 items covering domains including patient volume, available infrastructure, scope of practice, case distribution, and multidisciplinary care., Results: Responses from 196 individuals from 61 countries, spanning all WHO regions, were included. Ninety-six (49.0%) were from high-income countries, 57 (29.1%) were from upper-middle-income countries, 42 (21.4%) were from lower-middle-income countries (LMICs), and 1 was (0.5%) from a low-income country. Most respondents had a catchment population of ≥ 1 million and indicated the availability of basic neurosurgical resources such as a dedicated neurosurgical operating theater and surgical microscope. The presence of a neurosurgical intensive care unit, inpatient rehabilitation services, and infection monitoring showed similar availability across country groups. Quantitative scoring of 13 infrastructure and service items established that fewer resources were available in low-income countries/LMICs and upper-middle-income countries compared with high-income countries. The volume of pediatric CNS tumor cases and case distribution did not vary according to World Bank country groups., Conclusion: This study provides a comprehensive evaluation of pediatric neurosurgical capacity across the globe, establishing variability of resources on the basis of the country income level. Our findings suggest that pediatric neurosurgeons in LMICs may benefit from key neurosurgical instrumentation and increased support for multidisciplinary brain tumor programs and childhood cancer research efforts.
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- 2023
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3. Outcomes of Children With Low-Grade Gliomas in Low- and Middle-Income Countries: A Systematic Review.
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Ward R, Jones HM, Witt D, Boop F, Bouffet E, Rodriguez-Galindo C, Qaddoumi I, and Moreira DC
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- Child, Developing Countries, Humans, Retrospective Studies, Brain Neoplasms pathology, Brain Neoplasms surgery, Central Nervous System Neoplasms, Glioma pathology, Glioma surgery
- Abstract
Purpose: Pediatric CNS tumors are increasingly a priority, particularly with the WHO designation of low-grade glioma (LGG) as one of six index childhood cancers. There are currently limited data on outcomes of pediatric patients with LGGs in low- and middle-income countries (LMICs)., Methods: To better understand the outcomes of LGGs in LMICs, this systematic review interrogated nine literature databases., Results: The search identified 14,977 publications. Sixteen studies from 19 countries met the selection criteria and were included for data abstraction and analysis. Eleven studies (69%) were retrospective reviews from single institutions, and one (6%) captured institutional data prospectively. The studies captured a total of 957 patients with a median of 49 patients per study. Seven (44%) of the studies described the treatment modalities used. Of 373 patients for whom there was information, 173 (46%) had a gross total or near total resection, 109 (29%) had a subtotal resection, and 91 (24%) had only a biopsy performed. Seven studies, with a total of 476 patients, described the frequency of use of radiotherapy and/or chemotherapy in the cohorts: 83 of these patients received radiotherapy and 76 received chemotherapy. The 5-year overall survival ranged from 69.2% to 93.5%, although lower survival rates were reported at earlier time points. We identified limitations in the published studies with respect to the cohort sizes and methodologies., Conclusion: The included studies reported survival rates frequently exceeding 80%, although the ultimate number of studies was limited, pointing to the paucity of studies describing the outcomes of children with LGGs in LMICs. This study underscores the need for more robust data on outcomes in pediatric LGG.
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- 2022
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4. Cognitive and Adaptive Functioning in Youth With Retinoblastoma: A Longitudinal Investigation Through 10 Years of Age.
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Willard VW, Qaddoumi I, Pan H, Hsu CW, Brennan RC, Wilson MW, Rodriguez-Galindo C, Goode K, Parris K, and Phipps S
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- Child, Child, Preschool, Female, Humans, Infant, Infant, Newborn, Longitudinal Studies, Male, Cognition physiology, Intelligence Tests standards, Retinoblastoma epidemiology
- Abstract
Purpose: To describe the trajectory of cognitive and adaptive functioning in pediatric patients with retinoblastoma from diagnosis through age 10. This is an extension of a previous report that discussed findings from diagnosis through age 5., Patients and Methods: Ninety-eight participants with retinoblastoma completed psychological assessments as part of their enrollment on an institutional treatment protocol, with 73 completing an additional assessment at age 10. Trajectories of adaptive and cognitive functioning were determined, with data analyzed by treatment strata, and patients with 13q- analyzed separately., Results: Longitudinal trajectories identified a significant change point in trends at age 5, with functioning declining from diagnosis through age 5 and then increasing from age 5 to age 10. This pattern was observed for all strata for adaptive functioning, but only for enucleation-only patients (strata C low) for cognitive functioning. Cognitive trajectories were also influenced by laterality and enucleation status. At age 10, overall functioning was generally within the average range, although estimated intelligence quotient was significantly below the normative mean for enucleation-only (C low) patients. Patients with 13q- demonstrated very low functioning, but few analyses were significant because of small sample size., Conclusion: The results generally indicate that previously demonstrated declines in functioning from diagnosis through age 5 improve by age 10. However, these early declines, as well as the continuous difficulties observed in patients treated with enucleation only, suggest the need for early intervention services for young patients with retinoblastoma. Continuous monitoring of the psychological functioning of patients with retinoblastoma, increased awareness of risk factors such as unilateral disease, enucleation, race, and surgery-only treatment plans, and referral to Early Intervention for all patients are indicated., Competing Interests: Ibrahim QaddoumiConsulting or Advisory Role: SpringWorks Therapeutics, AstraZenecaNo other potential conflicts of interest were reported.
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- 2021
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5. Evaluation of the Pediatric Neuro-Oncology Resources Available in Chile.
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Abu-Arja MH, Rojas Del Río N, Morales La Madrid A, Lassaletta A, Coven SL, Moreno R, Valero M, Perez V, Espinoza F, Fernandez E, Santander J, Tordecilla J, Oyarce V, Kopp K, Bartels U, Qaddoumi I, Finlay JL, Cáceres A, Reyes M, Espinoza X, and Osorio DS
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- Child, Chile, Cross-Sectional Studies, Humans, Medical Oncology, Central Nervous System Neoplasms therapy, Hematopoietic Stem Cell Transplantation
- Abstract
Purpose: Pediatric neuro-oncology resources are mostly unknown in Chile. We report the human and material resources available in Chilean hospitals providing pediatric neuro-oncology services., Methods: A cross-sectional survey was distributed to 17 hospitals providing pediatric neuro-oncology services (Programa Infantil Nacional de Drogas Antineoplásicas [PINDA] hospitals, 11; private, 6)., Results: Response rate was 71% (PINDA, 8; private, 4). Pediatric neuro-oncology services were mainly provided within general hospitals (67%). Registries for pediatric CNS tumors and chemotherapy-related toxicities were available in 100% and 67% of hospitals, respectively. CNS tumors were treated by pediatric oncologists in 92% of hospitals; none were formally trained in neuro-oncology. The most used treatment protocols were the national PINDA protocols. All WHO essential medicines for childhood cancer were available in more than 80% of the hospitals except for gemcitabine, oxaliplatin, paclitaxel, and procarbazine. The median number of pediatric neurosurgeons per hospital was two (range, 2-6). General neuroradiologists were available in 83% of the centers. Pathology specimens were sent to neuropathologists (58%), adult pathologists (25%), and pediatric pathologists (17%). Intensity-modulated radiotherapy, conformal radiotherapy, and cobalt radiotherapy were used by 67%, 58%, and 42% of hospitals, respectively. Only one private hospital performed autologous hematopoietic cell transplant for children with CNS tumors., Conclusion: A wide range of up-to-date treatment modalities are available for children with CNS tumors. Our survey highlights future directions to improve the pediatric neuro-oncology services available in Chile such as the expansion of multidisciplinary clinics, palliative care services, long-term cancer survivorship programs, dedicated clinical research support teams, establishing standardized mechanism for sending pathologic specimen for second opinion to international specialized centers, and establishing specialized neuro-oncology training program.
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- 2021
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6. Assessment of Retinoblastoma Capacity in the Middle East, North Africa, and West Asia Region.
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Burges M, Qaddoumi I, Brennan RC, Krull L, Sahr N, Rodriguez-Galindo C, Jeha S, and Wilson MW
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- Africa, Northern, Asia, Child, Humans, Middle East epidemiology, Retinal Neoplasms, Retinoblastoma diagnosis, Retinoblastoma therapy
- Abstract
Purpose: We aimed to evaluate the capacity to treat retinoblastoma in the Middle East, North Africa, and West Asia region., Methods: A Web-based assessment that investigated retinoblastoma-related pediatric oncology and ophthalmology infrastructure and associated capacity at member institutions of the Pediatric Oncology East and Mediterranean group was distributed. Data were analyzed in terms of availability, location, and confidence of use for each resource needed for the management of retinoblastoma. Resources were categorized by diagnostics, focal therapy, chemotherapy, advanced treatment, and supportive care. Responding institutions were further divided into an asset-based tiered system., Results: In total, responses from 23 institutions were obtained. Fifteen institutions reported the availability of an ophthalmologist, 12 of which held primary off-site appointments. All institutions reported the availability of a pediatric oncologist and systemic chemotherapy A significant portion of available resources was located off site. Green laser was available on site at seven institutions, diode laser at six institutions, cryotherapy at 12 institutions, and brachytherapy at nine institutions. There existed marked disparity between the availability of some specific ophthalmic resources and oncologic resources., Conclusion: The assessment revealed common themes related to the treatment of retinoblastoma in low- and- middle-income countries, including decentralization of care, limited resources, and lack of multidisciplinary care. Resource disparities warrant targeted intervention in the Middle East, North Africa, and West Asia region to advance the management of retinoblastoma in the region.
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- 2020
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7. Bridging the Gap in Access to Care for Children With CNS Tumors Worldwide.
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Moreira DC, Rajagopal R, Navarro-Martin Del Campo RM, Polanco AC, Alcasabas AP, Arredondo-Navarro LA, Campos D, Casavilca-Zambrano S, Diaz Coronado RY, Faizal N, Ganesan D, Hamid SA, Khan R, Khu K, Loh J, Lukban M, Negreiros T, Pineda E, Yaqoob N, Zia N, Gajjar A, Rodriguez-Galindo C, and Qaddoumi I
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- Child, Humans, Central Nervous System Neoplasms therapy, Health Services Accessibility
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- 2020
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8. Global Access to Essential Medicines for Childhood Cancer: A Cross-Sectional Survey.
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Cohen P, Friedrich P, Lam C, Jeha S, Metzger ML, Qaddoumi I, Naidu P, Faughnan L, Rodriguez-Galindo C, and Bhakta N
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- Child, Cross-Sectional Studies, Female, Humans, Male, Neoplasms drug therapy
- Abstract
Purpose: Global data mapping access to essential chemotherapeutics for pediatric cancer are scarce. We report a survey of international pediatric cancer care providers' access to these medicines., Methods: A Web-based survey was sent to pediatric oncologists registered on the Cure4Kids Web portal. We queried chemotherapeutics in the WHO Essential Medicines List for Children, from which the average proportional availability was summarized as each country's access score. In addition, we examined availability of drug packages defined by the WHO-sanctioned Expert Committee for eight pediatric cancers. We undertook a sensitivity analysis investigating how regimen access would change if the cytotoxics specified in recent agreements between the Clinton Health Access Initiative, American Cancer Society, and pharmaceutical companies were universally available., Results: There were significant ( P < .001) differences in the median access scores between World Bank income groups, and 42.9% of respondents from low-income and lower middle-income countries reported suboptimal access scores. Our disease-based analysis revealed that 42.1% of patients in low-income and lower middle-income countries lacked full access to chemotherapy packages. Guaranteed availability of the cytotoxics specified in the Clinton Health Access Initiative/American Cancer Society agreements was projected to increase this regimen-based access by 1.6%, although including four additional chemotherapeutics would further increase coverage by 13.9%., Conclusion: This study is the first, to our knowledge, to assess worldwide variation in practical access to pediatric chemotherapy. Although mapping the proportion of available chemotherapeutics is informative, we also developed a meaningful estimate of access using disease-specific drug packages. These data provide an important baseline for continued monitoring and can aid in planning adaptive treatment guidelines that consider the trade-offs between access and outcomes.
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- 2018
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9. Video-Teleconferencing in Pediatric Neuro-Oncology: Ten Years of Experience.
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Amayiri N, Swaidan M, Abuirmeileh N, Al-Hussaini M, Tihan T, Drake J, Musharbash A, Qaddoumi I, Tabori U, Halalsheh H, Bartels U, and Bouffet E
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- Child, Developed Countries, Developing Countries, Humans, International Cooperation, Jordan, Oncologists, Ontario, Central Nervous System Neoplasms therapy, Videoconferencing
- Abstract
Purpose: The management of central nervous system tumors is challenging in low- and middle-income countries. Little is known about applicability of twinning initiatives with high-income countries in neuro-oncology. In 2004, a monthly neuro-oncology video-teleconference program was started between King Hussein Cancer Center (Amman, Jordan) and the Hospital for Sick Children (Toronto, Ontario, Canada). More than 100 conferences were held and > 400 cases were discussed. The aim of this work was to assess the sustainability of such an initiative and the evolution of the impact over time., Methods: We divided the duration in to three eras according to the initial 2 to 3 years of work of three consecutive oncologists in charge of the neuro-oncology program at King Hussein Cancer Center. We retrospectively reviewed the written minutes and compared the preconference suggested plans with the postconference recommendations. Impact of changes on the patient care was recorded., Results: Thirty-three sets of written minutes (covering 161 cases) in the middle era and 32 sets of written minutes (covering 122 cases) in the last era were compared with the initial experience (20 meetings, 72 cases). Running costs of these conferences has dropped from $360/h to < $40/h. Important concepts were introduced, such as multidisciplinary teamwork, second-look surgery, and early referral. Suggestions for plan changes have decreased from 44% to 30% and 24% in the respective consecutive eras. Most recommendations involved alternative intervention modalities or pathology review. Most of these recommendations were followed., Conclusion: Video-teleconferencing in neuro-oncology is feasible and sustainable. With time, team experience is built while the percentage and the type of treatment modifications change. Commitment and motivation helped maintain this initiative rather than availability of financial resources. Improvement in patients' care was achieved, in particular, with the implementation of a multidisciplinary team and the continuous effort to implement recommendations.
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- 2018
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10. Therapeutic and Prognostic Implications of BRAF V600E in Pediatric Low-Grade Gliomas.
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Lassaletta A, Zapotocky M, Mistry M, Ramaswamy V, Honnorat M, Krishnatry R, Guerreiro Stucklin A, Zhukova N, Arnoldo A, Ryall S, Ling C, McKeown T, Loukides J, Cruz O, de Torres C, Ho CY, Packer RJ, Tatevossian R, Qaddoumi I, Harreld JH, Dalton JD, Mulcahy-Levy J, Foreman N, Karajannis MA, Wang S, Snuderl M, Nageswara Rao A, Giannini C, Kieran M, Ligon KL, Garre ML, Nozza P, Mascelli S, Raso A, Mueller S, Nicolaides T, Silva K, Perbet R, Vasiljevic A, Faure Conter C, Frappaz D, Leary S, Crane C, Chan A, Ng HK, Shi ZF, Mao Y, Finch E, Eisenstat D, Wilson B, Carret AS, Hauser P, Sumerauer D, Krskova L, Larouche V, Fleming A, Zelcer S, Jabado N, Rutka JT, Dirks P, Taylor MD, Chen S, Bartels U, Huang A, Ellison DW, Bouffet E, Hawkins C, and Tabori U
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- Adolescent, Brain Neoplasms genetics, Brain Neoplasms pathology, Brain Neoplasms therapy, Brain Stem Neoplasms enzymology, Brain Stem Neoplasms genetics, Brain Stem Neoplasms pathology, Brain Stem Neoplasms therapy, Child, Child, Preschool, Cohort Studies, Diencephalon enzymology, Diencephalon pathology, Female, Glioma genetics, Glioma pathology, Glioma therapy, Humans, Infant, Male, Mutation, Neoplasm Grading, Prognosis, Brain Neoplasms enzymology, Glioma enzymology, Proto-Oncogene Proteins B-raf genetics
- Abstract
Purpose BRAF V600E is a potentially highly targetable mutation detected in a subset of pediatric low-grade gliomas (PLGGs). Its biologic and clinical effect within this diverse group of tumors remains unknown. Patients and Methods A combined clinical and genetic institutional study of patients with PLGGs with long-term follow-up was performed (N = 510). Clinical and treatment data of patients with BRAF V600E mutated PLGG (n = 99) were compared with a large international independent cohort of patients with BRAF V600E mutated-PLGG (n = 180). Results BRAF V600E mutation was detected in 69 of 405 patients (17%) with PLGG across a broad spectrum of histologies and sites, including midline locations, which are not often routinely biopsied in clinical practice. Patients with BRAF V600E PLGG exhibited poor outcomes after chemotherapy and radiation therapies that resulted in a 10-year progression-free survival of 27% (95% CI, 12.1% to 41.9%) and 60.2% (95% CI, 53.3% to 67.1%) for BRAF V600E and wild-type PLGG, respectively ( P < .001). Additional multivariable clinical and molecular stratification revealed that the extent of resection and CDKN2A deletion contributed independently to poor outcome in BRAF V600E PLGG. A similar independent role for CDKN2A and resection on outcome were observed in the independent cohort. Quantitative imaging analysis revealed progressive disease and a lack of response to conventional chemotherapy in most patients with BRAF V600E PLGG. Conclusion BRAF V600E PLGG constitutes a distinct entity with poor prognosis when treated with current adjuvant therapy.
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- 2017
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11. Ocular Salvage and Vision Preservation Using a Topotecan-Based Regimen for Advanced Intraocular Retinoblastoma.
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Brennan RC, Qaddoumi I, Mao S, Wu J, Billups CA, Stewart CF, Hoehn ME, Rodriguez-Galindo C, and Wilson MW
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- Antineoplastic Combined Chemotherapy Protocols adverse effects, Carboplatin administration & dosage, Child, Child, Preschool, Diarrhea chemically induced, Disease-Free Survival, Eye Enucleation, Female, Follow-Up Studies, Humans, Infant, Male, Radiotherapy, Retinoblastoma complications, Salvage Therapy, Survival Rate, Topotecan administration & dosage, Vincristine administration & dosage, Vision Disorders prevention & control, Visual Acuity, Antineoplastic Combined Chemotherapy Protocols therapeutic use, Hematologic Diseases chemically induced, Retinoblastoma therapy, Vision Disorders etiology
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Purpose To evaluate topotecan-based therapy for advanced intraocular retinoblastoma. Patients and Methods Twenty-seven patients with bilateral retinoblastoma (male patients, n = 14; median age, 8.4 months) received chemotherapy: two courses of topotecan plus vincristine followed by three alternating administrations of carboplatin and vincristine for two courses and topotecan plus vincristine for one course, with optional periocular carboplatin. Focal therapy was applied after cycle 2. Event-free survival was defined as avoidance of external-beam radiotherapy (EBRT) and enucleation. Results Of 54 eyes, 42 were Reese-Ellsworth group IV to V, and 37 were International Classification of Retinoblastoma group C to E. Eleven eyes were enucleated: one at diagnosis, nine with progressive disease including three eyes treated with EBRT, and one that developed neovascular glaucoma after completion of therapy. One patient was removed from protocol with prolonged infection in course 1; 26 patients (51 eyes) were analyzed. At 10 years, cumulative incidence of EBRT was 5.9% (SE ± 3), and event-free survival was 69.2% (SE ± 27.2). Ocular survival for Reese-Ellsworth group IV to V eyes was 77.5% (SE ± 21.2); it was 74.3% (SE ≥ 18.8) for International Classification group C to E eyes. Vision testing (median age, 7 years; range, 5 to 10 years) documented 20/70 vision or better in one eye of 23 patients; 19 (76%) of 25 patients demonstrated 20/40 vision or better in one eye. All patients experienced thrombocytopenia (41 episodes in 275 courses; 15%). There were 29 episodes of febrile neutropenia (10%). Grade 3 diarrhea was present in nine of 27 patients, and one patient had an allergic reaction to carboplatin. All patients are alive at median follow-up 7.4 years (range, 2.7 to 10 years). Conclusion Topotecan combined with vincristine, carboplatin, and aggressive focal therapies is an effective regimen for the treatment of advanced retinoblastoma and results in globe salvage with vision. Toxicities were anticipated and managed with appropriate supportive care.
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- 2017
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12. Challenges of Treating Childhood Medulloblastoma in a Country With Limited Resources: 20 Years of Experience at a Single Tertiary Center in Malaysia.
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Rajagopal R, Abd-Ghafar S, Ganesan D, Bustam Mainudin AZ, Wong KT, Ramli N, Jawin V, Lum SH, Yap TY, Bouffet E, Qaddoumi I, Krishnan S, Ariffin H, and Abdullah WA
- Abstract
Purpose: Pediatric medulloblastoma (MB) treatment has evolved over the past few decades; however, treating children in countries with limited resources remains challenging. Until now, the literature regarding childhood MB in Malaysia has been nonexistent. Our objectives were to review the demographics and outcome of pediatric MB treated at the University Malaya Medical Center between January 1994 and December 2013 and describe the challenges encountered., Methods: Fifty-one patients with childhood MB were seen at University Malaya Medical Center. Data from 43 patients were analyzed; eight patients were excluded because their families refused treatment after surgery., Results: Headache and vomiting were the most common presenting symptoms, and the mean interval between symptom onset and diagnosis was 4 weeks. Fourteen patients presented with metastatic disease. Five-year progression-free survival (± SE) for patients ≥ 3 years old was 41.7% ± 14.2% (95% CI, 21.3% to 81.4%) in the high-risk group and 68.6% ± 18.6% (95% CI, 40.3% to 100%) in the average-risk group, and 5-year overall survival (± SE) in these two groups was 41.7% ± 14.2% (95% CI, 21.3% to 81.4%) and 58.3% ± 18.6% (95% CI, 31.3% to 100%), respectively. Children younger than 3 years old had 5-year progression-free and overall survival rates (± SE) of 47.6% ± 12.1% (95% CI, 28.9% to 78.4%) and 45.6% ± 11.7% (95% CI, 27.6% to 75.5%), respectively. Time to relapse ranged from 4 to 132 months. Most patients who experienced relapse died within 1 year. Febrile neutropenia, hearing loss, and endocrinopathy were the most common treatment-related complications., Conclusion: The survival rate of childhood MB in Malaysia is inferior to that usually reported in the literature. We postulate that the following factors contribute to this difference: lack of a multidisciplinary neuro-oncology team, limited health care facilities, inconsistent risk assessment, insufficient data in the National Cancer Registry and pathology reports, inadequate long-term follow-up, and cultural beliefs leading to treatment abandonment., Competing Interests: The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/jco/site/ifc. Revathi RajagopalNo relationship to discloseSayyidatul Abd-GhafarNo relationship to discloseDharmendra GanesanNo relationship to discloseAnita Zarina Bustam MainudinTravel, Accommodations, Expenses: MSD OncologyKum Thong WongNo relationship to discloseNorlisah RamliNo relationship to discloseVida JawinNo relationship to discloseSu Han LumNo relationship to discloseTsiao Yi YapNo relationship to discloseEric BouffetResearch Funding: GenentechIbrahim QaddoumiNo relationship to discloseShekhar KrishnanTravel, Accommodations, Expenses: Sigma-TauHany AriffinNo relationship to discloseWan Ariffin AbdullahNo relationship to disclose
- Published
- 2016
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13. Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032.
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Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, Packer RJ, Goldman S, Prados MD, Desjardins A, Chintagumpala M, Takebe N, Kaste SC, Rusch M, Allen SJ, Onar-Thomas A, Stewart CF, Fouladi M, Boyett JM, Gilbertson RJ, Curran T, Ellison DW, and Gajjar A
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- Adult, Brain Neoplasms genetics, Brain Neoplasms metabolism, Female, Hedgehog Proteins genetics, Humans, Male, Medulloblastoma genetics, Medulloblastoma metabolism, Middle Aged, Young Adult, Anilides therapeutic use, Brain Neoplasms drug therapy, Hedgehog Proteins metabolism, Medulloblastoma drug therapy, Pyridines therapeutic use
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Purpose: Two phase II studies assessed the efficacy of vismodegib, a sonic hedgehog (SHH) pathway inhibitor that binds smoothened (SMO), in pediatric and adult recurrent medulloblastoma (MB)., Patients and Methods: Adult patients enrolled onto PBTC-025B and pediatric patients enrolled onto PBTC-032 were treated with vismodegib (150 to 300 mg/d). Protocol-defined response, which had to be sustained for 8 weeks, was confirmed by central neuroimaging review. Molecular tests to identify patterns of response and insensitivity were performed when tissue was available., Results: A total of 31 patients were enrolled onto PBTC-025B, and 12 were enrolled onto PBTC-032. Three patients in PBTC-025B and one in PBTC-032, all with SHH-subgroup MB (SHH-MB), exhibited protocol-defined responses. Progression-free survival (PFS) was longer in those with SHH-MB than in those with non-SHH-MB, and prolonged disease stabilization occurred in 41% of patient cases of SHH-MB. Among those with SHH-MB, loss of heterozygosity of PTCH1 was associated with prolonged PFS, and diffuse staining of P53 was associated with reduced PFS. Whole-exome sequencing identified mutations in SHH genes downstream from SMO in four of four tissue samples from nonresponders and upstream of SMO in two of four patients with favorable responses., Conclusion: Vismodegib exhibits activity against adult recurrent SHH-MB but not against recurrent non-SHH-MB. Inadequate accrual of pediatric patients precluded conclusions in this population. Molecular analyses support the hypothesis that SMO inhibitor activity depends on the genomic aberrations within the tumor. Such inhibitors should be advanced in SHH-MB studies; however, molecular and genomic work remains imperative to identify target populations that will truly benefit., (© 2015 by American Society of Clinical Oncology.)
- Published
- 2015
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14. Developmental and adaptive functioning in children with retinoblastoma: a longitudinal investigation.
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Willard VW, Qaddoumi I, Chen S, Zhang H, Brennan R, Rodriguez-Galindo C, Wilson MW, and Phipps S
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- Child, Child, Preschool, Cognition physiology, Female, Humans, Infant, Infant, Newborn, Longitudinal Studies, Male, Retinal Neoplasms psychology, Retinoblastoma psychology, Adaptation, Physiological physiology, Retinal Neoplasms physiopathology, Retinoblastoma physiopathology
- Abstract
Purpose: To determine the developmental trajectory of early cognitive and adaptive skills in young children with retinoblastoma from diagnosis to 5 years of age., Patients and Methods: Ninety-four patients with retinoblastoma treated according to an institutional protocol underwent serial assessments of cognitive and adaptive functioning at age 6 months and 1, 2, 3, and 5 years. Data were analyzed by treatment strata, with patients with 13q deletion analyzed separately., Results: At baseline, across all patients (except those with 13q deletion), developmental functioning was comparable with the normative mean, with mean scores for all strata within the average range. However, at age 5 years, developmental functioning was in the low average range and significantly below normative means. The trajectories of developmental functioning demonstrated significant decline over time, although this varied by treatment group/strata. Patients treated with enucleation only evidenced the greatest decline in cognitive functioning; significant change was not observed in patients treated with other modalities. Notable declines in parent-reported communication skills were observed in the majority of patients. Patients with 13q deletion evidenced delayed cognitive functioning at baseline, but minimal declines were observed through age 3 years. However, significant decreases in adaptive functioning were demonstrated over time for the 13q deletion subset., Conclusion: The declines in functioning observed in this study were unexpected, as was the poorer performance of the enucleation-only group. This highlights the necessity of continuing to assess cognitive functioning in patients with retinoblastoma as they age. Additional research is necessary to determine the long-term trajectory of cognitive development in this population., (© 2014 by American Society of Clinical Oncology.)
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- 2014
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15. Carboplatin-associated ototoxicity in children with retinoblastoma.
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Qaddoumi I, Bass JK, Wu J, Billups CA, Wozniak AW, Merchant TE, Haik BG, Wilson MW, and Rodriguez-Galindo C
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- Antineoplastic Combined Chemotherapy Protocols administration & dosage, Carboplatin administration & dosage, Dose-Response Relationship, Drug, Drug Administration Schedule, Female, Hearing Loss epidemiology, Hearing Tests, Humans, Incidence, Infant, Male, Retrospective Studies, Risk Factors, Severity of Illness Index, Vincristine administration & dosage, Antineoplastic Combined Chemotherapy Protocols adverse effects, Carboplatin adverse effects, Hearing drug effects, Hearing Loss chemically induced, Retinal Neoplasms drug therapy, Retinoblastoma drug therapy
- Abstract
Purpose: Carboplatin-induced ototoxicity remains poorly defined but is of potential great consequence in children with retinoblastoma. We retrospectively assessed the incidence of ototoxicity and its risk factors in children with retinoblastoma who were treated with carboplatin., Patients and Methods: We reviewed the audiologic test results of 60 patients with retinoblastoma who received front-line treatment with systemic carboplatin and vincristine according to the St Jude RET-3 protocol (n = 23) or best clinical management (n = 37). Ototoxicity was evaluated by three different grading systems., Results: Twelve patients (20%) developed ototoxicity at some time after treatment initiation; however, ototoxicity resolved in two patients, and thus,10 patients (17%) had sustained hearing loss as documented at their most recent audiologic evaluation. Nine of these 10 patients had grade 3 or 4 ototoxicity, and nine patients were less than 6 months of age at the start of chemotherapy. Age at the start of chemotherapy was the only risk factor identified as a significant predictor of sustained hearing loss. Younger age was associated with an increased incidence of hearing loss. The different ototoxicity grading systems showed good overall agreement in the identification of patients with ototoxicity. Agreement was greatest between the Brock and Children's Cancer Group systems., Conclusion: We found that young patients with retinoblastoma who were treated with systemic carboplatin had a higher incidence of ototoxicity than previously reported. Younger patients (< 6 months of age at the start of treatment) were more likely to have ototoxicity than were older patients. Children treated with carboplatin should routinely undergo thorough, long-term audiologic monitoring.
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- 2012
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16. Is it pre-enucleation chemotherapy or delayed enucleation of severely involved eyes with intraocular retinoblastoma that risks extraocular dissemination and death?
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Chantada G, Leal-Leal C, Brisse H, de Graaf P, Sitorus RS, Qaddoumi I, de Antoneli CB, Tacyildiz N, Fineman SL, Lumbroso L, and Doz F
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- Female, Humans, Male, Antineoplastic Agents adverse effects, Neoadjuvant Therapy adverse effects, Premedication methods, Retinal Neoplasms drug therapy, Retinal Neoplasms mortality, Retinoblastoma mortality, Retinoblastoma secondary
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- 2011
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17. Comparing adult and pediatric rhabdomyosarcoma in the surveillance, epidemiology and end results program, 1973 to 2005: an analysis of 2,600 patients.
- Author
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Sultan I, Qaddoumi I, Yaser S, Rodriguez-Galindo C, and Ferrari A
- Subjects
- Adolescent, Adult, Age Distribution, Aged, Aged, 80 and over, Child, Child, Preschool, Female, Humans, Infant, Infant, Newborn, Kaplan-Meier Estimate, Male, Middle Aged, Multivariate Analysis, Population Surveillance, Prognosis, Proportional Hazards Models, Rhabdomyosarcoma diagnosis, Rhabdomyosarcoma therapy, United States epidemiology, Young Adult, Rhabdomyosarcoma epidemiology, SEER Program statistics & numerical data
- Abstract
Purpose: To compare clinical features and outcomes of adults and children reported to have rhabdomyosarcoma., Patients and Methods: We analyzed data from 1,071 adults (age > 19 years) and 1,529 children (age < or = 19 years) reported in the public-access Surveillance, Epidemiology and End Results database as having rhabdomyosarcoma, diagnosed from 1973 to 2005. Survival estimates were determined using survival time with the end point being death from any cause., Results: Adults with rhabdomyosarcoma had significantly worse outcome than children (5-year overall survival rates, 27% +/- 1.4% and 61% +/- 1.4%, respectively; P < .0001). Tumors in adults were more likely to be at an unfavorable site (65% v 55%; P < .0001) and to have histologies that are unusual during childhood, particularly the pleomorphic subtype (19%) and not otherwise specified (43%). Regional and distant spread was not more frequent in adults. Adults had significantly worse outcome than children with similar tumors. The most significant difference was in localized disease; 5-year survival estimates were 82% +/- 2.0% for children and 47% +/- 2.9% for adults (P < .0001). Multivariate analysis showed that age, histologic subtype, primary site location, stage, and local control with surgery and/or radiation were significant predictors of survival. However, alveolar subtype and unfavorable primary site lost significance when analysis was restricted to adults., Conclusion: Adults reported to have rhabdomyosarcoma had worse survival than children with similar tumors. Predictors of poor outcome in children were valid in adults except for alveolar histology and unfavorable tumor site.
- Published
- 2009
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