EPID-21. THE NATIONAL SPECTRUM OF NEWLY-DIAGNOSED BRAIN TUMORS IN ADULT PATIENTS VARIES SIGNIFICANTLY BY PATIENT DEMOGRAPHICS

Abstract INTRODUCTION Herein we examine the epidemiology and outcomes of the entire spectrum of intracranial tumors in the contemporary era. METHODS Adult patients (≥20yo) presenting between 2010–2015 where the first evidence of cancer involves an intracranial tumor were queried from the National Cancer Database, which comprises >70% of cancers newly-diagnosed in the U.S. Tumor types were classified by WHO2016 ICD-O3, and stratified by patient characteristics. RESULTS 361,841 adults without a history of cancer presented with intracranial tumors between 2010–2015. Across all ages, these were comprised of 1) brain metastases (BMs; 25%: 29% in males vs. 23% in females); 2) meningiomas (25%: 15% in males, but 34% in females) including atypical (n=4,565) and anaplastic (n=833); 3) diffuse infiltrative gliomas (21%: 26% in males vs. 17% in females), mostly GBMs (14%); followed by 4) sellar (14%), 5) cranial nerve (6%), and PCNSL (3%) tumors. The remaining types each comprised ≤2 % of brain tumors, including mesenchymal non-meningothelial tumors (2%), intracranial ependymal (0.8%), mixed neuronal-glial (0.7%), circumscribed “other astrocytomas” (0.6%, mostly pilocytic astrocytomas n=1,307 and PXAs n=272), CNS embryonal (0.3%), pineal (0.2%), GCTs (0.1%), and choroid plexus (0.1%) tumors. In the 91,686 patients presenting with a BM, the most common primaries were lung adenocarcinoma (39%), lung SmallCC (14%), lung SqCC (8%), breast (8% of females), melanoma (3%), kidney (3%), colorectal (2%), and esophageal (1%). The distributions of brain tumor types differed significantly by age, sex, race/ethnicity, and insurance status. CONCLUSIONS In adult patients where the first manifestation of cancer includes an intracranial tumor, the most common diagnosis is either metastatic disease (predominantly from NSCLC) in males or benign meningiomas in females; but varies substantially by age group. Notably, our results adjust the traditional teaching that half of all new brain masses are BMs, which in fact represent only ~25% of new intracranial masses.

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Cerebellar liponeurocytoma: a rare intracranial tumor with possible familial predisposition. Case report

Journal of Neurosurgery ◽

10.3171/2015.6.jns142965 ◽

2016 ◽

Vol 125 (1) ◽

pp. 57-61 ◽

Cited By ~ 5

Author(s):

Amparo Wolf ◽

Huda Alghefari ◽

Daria Krivosheya ◽

Michael D. Staudt ◽

Gregory Bowden ◽

...

Keyword(s):

Tumor Development ◽

Healthy Woman ◽

Intracranial Tumor ◽

Intracranial Tumors ◽

Histopathological Changes ◽

Biological Origin ◽

Familial Predisposition ◽

History Of ◽

Cerebellar Liponeurocytoma ◽

Supracerebellar Infratentorial Approach

The biological origin of cerebellar liponeurocytomas is unknown, and hereditary forms of this disease have not been described. Here, the authors present clinical and histopathological findings of a young patient with a cerebellar liponeurocytoma who had multiple immediate family members who harbored similar intracranial tumors. A 37-year-old otherwise healthy woman presented with a history of progressive headaches. Lipomatous medulloblastoma had been diagnosed previously in her mother and maternal grandfather, and her maternal uncle had a supratentorial liponeurocytoma. MRI revealed a large, poorly enhancing, lipomatous mass emanating from the superior vermis that produced marked compression of posterior fossa structures. An uncomplicated supracerebellar infratentorial approach was used to resect the lesion. Genetic and histopathological analyses of the lesion revealed neuronal, glial, and lipomatous differentiation and confirmed the diagnosis of cerebellar liponeurocytoma. A comparison of the tumors resected from the patient and, 22 years previously, her mother revealed similar features. Cerebellar liponeurocytoma is a poorly understood entity. This report provides novel evidence of an inheritable predisposition for tumor development. Accurate diagnosis and reporting of clinical outcomes and associated genetic and histopathological changes are necessary for guiding prognosis and developing recommendations for patient care.

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OTHR-10. THE NATIONAL DISTRIBUTION OF NEWLY-DIAGNOSED BRAIN METASTASES IN ADULTS VARIES WIDELY BY PATIENT DEMOGRAPHICS

Neuro-Oncology Advances ◽

10.1093/noajnl/vdz014.087 ◽

2019 ◽

Vol 1 (Supplement_1) ◽

pp. i20-i20

Author(s):

Vasileios Kavouridis ◽

Matthew Torre ◽

Maya Harary ◽

Timothy Smith ◽

Bryan Iorgulescu

Keyword(s):

Brain Metastases ◽

Pacific Islanders ◽

Prior History ◽

National Cancer Database ◽

Newly Diagnosed ◽

Patient Demographics ◽

Asian Pacific Islanders ◽

History Of ◽

Asian Pacific ◽

History Of Cancer

Abstract INTRODUCTION: Metastases are oft-cited as comprising approximately half of all adult intracranial neoplasms, and their national composition remains unclear. METHODS: The patient demographics and histologic distribution of newly-diagnosed brain metastasis (BM) patients aged > 18yo without a prior history of cancer (2010–2015) were evaluated using the National Cancer Database, which comprises > 70% of all newly-diagnosed cancers in the U.S. RESULTS: 91,686 adults presented with a newly-diagnosed BM between 2010–2015. The most common sites of brain metastases overall were lung (82% of metastatic cases), breast (4.1%), melanoma (3.2%), kidney (2.9%), and colorectal (1.8%). The overall 1-year and 5-year OS rates for all BMs were 27.0% (95% CI [26.7%-27.3%]) and 5.3% (95% CI [5.1%-5.5%]), respectively. The distribution of primary sites for newly-diagnosed BMs varied by sex, age, and race. Compared to males, more females had BMs from breast (8.4% versus 0.8%) and fewer had BMs from kidney (1.9% versus 3.8%), melanoma (1.9% versus 4.5%), and esophagus (0.3% versus 2.0%). In young adults, particularly those 20-29yo, BMs were more likely from melanoma, genitourinary (in males), and soft tissue than adults in middle and advanced age. Lung carcinomas comprised fewer BMs in Hispanics (66%) compared to Whites (82%), Blacks (83%), and Asian/Pacific Islanders (85%). BMs from kidney and genitourinary primaries were higher in Hispanics (7.3% and 2.4% of BMs, respectively) than in Whites (2.8% and 0.3%, respectively), Blacks (1.8% and 0.1%, respectively), and Asian/Pacific Islanders (2.6% and 0.2%, respectively). Melanoma was more frequent in Whites (3.8% of BMs) and Hispanics (2.5%) compared to Blacks (0.3%) and Asian/Pacific Islanders (0.6%). CONCLUSION: Our results illustrate the national distribution of newly-diagnosed BMs and investigates how the distribution varies by patient demographics.

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The long-term side effects of radiation therapy for benign brain tumors in adults

Journal of Neurosurgery ◽

10.3171/jns.1990.73.4.0502 ◽

1990 ◽

Vol 73 (4) ◽

pp. 502-512 ◽

Cited By ~ 245

Author(s):

Ossama Al-Mefty ◽

Jane E. Kersh ◽

Anupam Routh ◽

Robert R. Smith

Keyword(s):

Radiation Therapy ◽

Side Effects ◽

Brain Tumors ◽

Adult Patients ◽

Intracranial Tumors ◽

Content Type ◽

Pituitary Dysfunction ◽

Fine Print

✓ Radiation therapy plays an integral part in managing intracranial tumors. While the risk:benefit ratio is considered acceptable for treating malignant tumors, risks of long-term complications of radiotherapy need thorough assessment in adults treated for benign tumors. Many previously reported delayed complications of radiotherapy can be attributed to inappropriate treatment or to the sensitivity of a developing child's brain to radiation. Medical records, radiological studies, autopsy findings, and follow-up information were reviewed for 58 adult patients (31 men and 27 women) treated between 1958 and 1987 with radiotherapy for benign intracranial tumors. Patient ages at the time of irradiation ranged from 21 to 87 years (mean 47.7 years). The pathology included 46 pituitary adenomas, five meningiomas, four glomus jugulare tumors, two pineal area tumors, and one craniopharyngioma. Average radiation dosage was 4984 cGy (range 3100 to 7012 cGy), given in an average of 27.2 fractions (range 15 to 45 fractions), over a period averaging 46.6 days. The follow-up period ranged from 3 to 31 years (mean 8.1 years). Findings related to tumor recurrence or surgery were excluded. Twenty-two patients had complications considered to be delayed side effects of radiotherapy. Two patients had visual deterioration developing 3 and 6 years after treatment; six had pituitary dysfunction; and 17 had varying degrees of parenchymal changes of the brain, occurring mostly in the temporal lobes and relating to the frequent presentation of pituitary tumors (two of these also had pituitary dysfunction). One clival tumor, with the radiographic appearance of a meningioma, developed 30 years post-irradiation for acromegaly. This study unveils considerable delayed sequelae of radiotherapy in a series of adult patients receiving what is considered “safe” treatment for benign brain tumors.

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Treatment-Related Morbidity and Mortality Associated with Remission Induction in Adolescent and Young Adult (AYA) Versus Young Adult Patients with Newly Diagnosed Acute Myeloid Leukemia (AML)

Blood ◽

10.1182/blood.v128.22.5167.5167 ◽

2016 ◽

Vol 128 (22) ◽

pp. 5167-5167

Author(s):

Cory Michael Vela ◽

Viet Q. Ho ◽

Justina Ofori Frimpong ◽

Bryan Brinda ◽

Timothy George ◽

...

Keyword(s):

Young Adult ◽

Induction Chemotherapy ◽

Research Funding ◽

Univariate Analysis ◽

Adult Patients ◽

Morbidity And Mortality ◽

Newly Diagnosed ◽

Hematologic Disease ◽

History Of ◽

Baseline Characteristics

Abstract Background: Acute myeloid leukemia is the most commonly diagnosed acute leukemia with a median age of diagnosis between 65 - 70 years; 45% of newly diagnosed AML patients are less than 65 years of age. Complete response (CR) rates following induction chemotherapy ranges between 60 - 80% in patients younger than 60 years old. However, limited data exists evaluating AML outcomes of adolescent and young adult (AYA), defined as 15 - 39 years, compared to adult patients less than 60 years old. Herein, we compare treatment-related outcomes of AYA versus adult AML patients. Methods: This was a single-center, retrospective study comparing treatment-related outcomes, morbidity and mortality between newly diagnosed AYA and adult AML patients receiving induction chemotherapy from July 1, 2009 to July 31, 2014 treated at Moffitt Cancer Center. Patients were excluded if less than 18 years of age, diagnoses of acute promyelocytic leukemia (APL) or myelodysplastic syndrome (MDS). The primary objective was to compare 30-day mortality between the AYA and adult AML patients admitted to MCC. Secondary objectives included 60-day mortality, death during induction, CR rates, overall survival (OS), receipt of hematopoietic stem-cell transplant (HSCT), infection, transfer to the intensive care unit (ICU), complications, organ failure, and chemotherapy toxicity. Results: A total of 176 patients were identified, of which 47 were aged 18 - 39 and 129 were aged 40 - 59. Baseline characteristics were similar among AYA and adult patients except for age, weight, body mass index, obesity, and those with greater than 3 comorbidities. Thirty-day mortality rates between AYA and adult patients were similar (2% vs 3%, p=0.596). Additionally, 60-day mortality (2% vs 6%), death during induction hospitalization (2% vs 5.5%), CR rates (70% vs 72%), receipt of HSCT (42.5% vs 47%), median OS (Not Reached vs 25.18 months, Figure 1), ICU transfer (10.5% vs 17%), time to infection (15.5 days vs 16 days), rate of infections (34% vs 33%), time to complication (8 days vs 10 days), rate of organ failure, and rate of chemotherapy toxicity were not significantly different between the two groups. Number of patients experiencing a treatment-related complication was significantly lower in AYA versus adult patients (74.5% vs 94%, p<0.05). Univariate analysis found AYA age group, receipt of HSCT, history of antecedent hematologic disease, and cytogenetic risk factor as significant variables impacting OS. A multivariate analysis identified cytogenetic risk factor (OR, 2.045; 95% CI 1.409 - 2.969), history of antecedent hematologic disease (OR, 1.635; 95% CI 1.018 - 2.628), and receipt of HSCT (OR, 0.397; 95% CI 0.254 - 0.62) as predictors for OS. Conclusion: Overall, there were no significant differences in treatment-related morbidity and mortality between AYA and adult newly diagnosed AML patients who received induction chemotherapy. Patients receiving HSCT, history of antecedent hematologic disease, and cytogenetic risk category showed an effect on multivariate analysis of OS while AYA age group was only significant in the univariate analysis. Figure 1 OS Curve of AYA vs Adult Patients Figure 1. OS Curve of AYA vs Adult Patients Figure 2 Baseline Characteristics Figure 2. Baseline Characteristics Disclosures Sweet: Incyte Corporation: Research Funding; Karyopharm: Honoraria, Research Funding; Pfizer: Speakers Bureau; Novartis: Consultancy, Speakers Bureau; Ariad: Consultancy, Speakers Bureau. Komrokji:Novartis: Consultancy, Speakers Bureau; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding.

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Pediatric Stapes Surgery: Hearing and Surgical Outcomes in Endoscopic vs Microscopic Approaches

Otolaryngology ◽

10.1177/0194599819836679 ◽

2019 ◽

Vol 161 (1) ◽

pp. 150-156 ◽

Cited By ~ 7

Author(s):

Anthony M. Tolisano ◽

Miles R. Fontenot ◽

Ashley M. Nassiri ◽

Jacob B. Hunter ◽

Joe Walter Kutz ◽

...

Keyword(s):

Case Series ◽

Stapes Surgery ◽

Middle Ear Surgery ◽

Patient Demographics ◽

Common Diagnosis ◽

Stapes Footplate ◽

History Of ◽

Revision Stapes Surgery ◽

Hearing Outcomes ◽

Endoscopic Group

Objective To compare endoscopic and microscopic pediatric stapes surgery. Study Design Case series with chart review. Setting Two academic otology practices. Subjects and Methods Surgical and hearing outcomes were compared for consecutive children (<18 years) undergoing microscopic and endoscopic stapes surgery. The main outcome measure was closure of the air-bone gap (ABG) to ≤20 dB. Results Twenty-two endoscopic surgeries (17 stapedectomies, 4 stapedotomies, and 1 stapes mobilization) and 52 microscopic surgeries (30 stapedectomies, 19 stapedotomies, and 3 stapes mobilizations) were performed. Patient demographics, history of ipsilateral middle ear surgery, and revision stapes surgery status were similar. The most common diagnosis for the endoscopic group and microscopic group were congenital stapes footplate fixation (45.5%) and juvenile otosclerosis (46.2%), respectively. Preoperative ABGs in the endoscopic (37.7 dB) and microscopic (32.8 dB) groups ( P = .170) were similar. There were no major complications, including facial nerve injury or anacusis, in the endoscopic group. Postoperative sensorineural hearing loss (>15 dB) did not occur in any patients in the endoscopic group but was present in 2 patients in the microscopic group ( P = .546). Improvement in pure-tone average (25.9 dB vs 18.5 dB, P = .382) and ABG (21.7 dB vs 14.7 dB, P = .181) was similar, and postoperatively, the median ABG was 11.3 dB and 15.0 dB for endoscopic and microscopic cases ( P = .703), respectively. ABG closure to ≤20 dB (72.7% vs 65.2%, P = .591) was also similar. Conclusion Pediatric endoscopic stapes surgery is safe and hearing outcomes are similar to the microscopic approach when performed by experienced endoscopic ear surgeons.

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EPID-22. NEWLY-DIAGNOSED BRAIN TUMORS IN PEDIATRIC PATIENTS: EPIDEMIOLOGY IN THE UNITED STATES

Neuro-Oncology ◽

10.1093/neuonc/noz175.322 ◽

2019 ◽

Vol 21 (Supplement_6) ◽

pp. vi79-vi79

Author(s):

Matthew Torre ◽

Mustafa Ascha ◽

Maya Harary ◽

Timothy Smith ◽

Ayal Aizer ◽

...

Keyword(s):

Pediatric Patients ◽

The United States ◽

Pineal Region ◽

Intracranial Tumor ◽

Astrocytic Tumors ◽

Intracranial Tumors ◽

Newly Diagnosed ◽

Embryonal Tumors ◽

Diffuse Gliomas ◽

The U.S

Abstract INTRODUCTION Herein we examine the epidemiology across all pediatric intracranial tumors in the U.S. METHODS Pediatric patients (< 20yo) presenting between 2010–2015 with an intracranial tumor as the first evidence of cancer were queried from the National Cancer Database, which comprises >70% of cancers newly-diagnosed in the U.S. Tumor types were classified by WHO2016 ICD-O3. RESULTS 14,952 pediatric patients without a history of cancer presented with intracranial tumors between 2010–2015. Across all ages, these were comprised of 1) 22% diffuse astrocytic and oligodendroglial (n=3,328), 2) 17% sellar region (n=2,530), 3) 16% other astrocytic (n=2,378, 2,131 were pilocytic and pilomyxoid), 4) 13% embryonal (n=1,896; 1,092 were classic histology medulloblastomas), 5) 7% neuronal and mixed neuronal-glial (n=981), 6) 5% ependymal (n=752), 7) 4% GCTs (n=615), 8) 3% mesenchymal non-meningothelial (n=492), 9) 3% nerve sheath (n=428), and 10) ≤2% each of meningioma, choroid plexus, brain metastatic (44% from neuroblastoma), pineal region, hematologic, and other glioma tumors. Embryonal tumors predominated in neonates and infants (0-2yo), diffuse gliomas in childhood (3-11yo), and sellar tumors in adolescents (12-19yo). Females represented 49% of the cases, but sellar tumors predominated (23%, vs. only 11% in males); whereas in males diffuse gliomas predominated (23%; vs. 22% in females) overall and in pediatric patients older than 2yo. CONCLUSIONS In U.S. pediatric patients that present with a new intracranial tumor, embryonal tumors and diffuse gliomas predominate in infants ≤2yo, and continue to predominate in male children 3-11yo, whereas female children 3-11yo shifted towards a predominance by diffuse gliomas and other astrocytic tumors (e.g. pilocytic and pilomyxoid). In female adolescents 12-19yo, sellar tumors predominated, followed by diffuse gliomas; whereas in male adolescents diffuse gliomas predominated, followed by other astrocytic tumors and sellar tumors. Our findings demonstrate that newly-diagnosed intracranial masses in pediatric patients significantly vary by both age and sex.

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Headache in Intracranial Tumors

Cephalalgia ◽

10.1046/j.1468-2982.1999.1909787.x ◽

1999 ◽

Vol 19 (9) ◽

pp. 787-790 ◽

Cited By ~ 78

Author(s):

Z Pfund ◽

L Szapáry ◽

O Jászberényi ◽

F Nagy ◽

J Czopf

Keyword(s):

Intracranial Pressure ◽

Brain Tumors ◽

Intracranial Tumors ◽

Metastatic Brain Tumors ◽

Increased Intracranial Pressure ◽

Close Relationship ◽

History Of ◽

Intraventricular Tumors ◽

Intracranial Neoplasm ◽

Headache Patients

The clinical data of 279 consecutive patients with brain tumors were analyzed pre- and postoperatively in the period of 1994-95. No headache had been recorded in the history of 115 patients, neither pre- nor postoperatively. Only in 139 of the remaining 164 headache patients was there a probable connection between headache and intracranial neoplasm. In the headache group the most frequent findings were metastatic brain tumors and different astrocytomas. Hypophysis adenomas and glioblastoma multiforme were frequent in the no-headache group. Progressive headache was found in 110 patients (67% of the headache group). The progressive character of the headache showed a close relationship with the prevailing edema, but not with the size of the tumor. Infratentorial and intraventricular tumors were more frequently accompanied by headache than those located supratentorially, probably due to the disturbance of CSF circulation and midline dislocation with increased intracranial pressure. Only in one-third of the patients did the site of the tumor coincide with the lateralization of headache. In half of the headache patients, pain was the first complaint. Headaches caused by tumor were characterized by pain lasting for hours, developing for weeks or months. The headache was never permanent and there was no regular daily recurrence.

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A Phase II Study to Investigate the Efficacy and Safety of Eltrombopag in Combination with Dexamethasone As First-Line Treatment in Adult Patients with Newly Diagnosed Primary ITP (XPAG-ITP)

Blood ◽

10.1182/blood-2020-134318 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 36-37

Author(s):

Axel Matzdorff ◽

Mascha Binder ◽

Falk Nimmerjahn ◽

Oliver Meyer ◽

Mathias J. Rummel ◽

...

Keyword(s):

Treatment Discontinuation ◽

Sustained Response ◽

Adult Patients ◽

High Dose ◽

Newly Diagnosed ◽

Platelet Counts ◽

High Dose Dexamethasone ◽

Short Course ◽

Proliferation And Differentiation ◽

History Of

BACKGROUND: Eltrombopag is an oral, small-molecule, non-peptide thrombopoietin receptor agonist (TPO-RA) that increases hematopoiesis by inducing proliferation and differentiation of early bone marrow progenitor cells leading to increased platelet production (Erickson-Miller et al., 2010, Sun et al., 2012). Eltrombopag has demonstrated efficacy in adult and pediatric patients with immune thrombocytopenia (ITP) and has a well-established safety profile. It is approved in Europe for the treatment of thrombocytopenia, from 6 months following diagnosis, in patients with primary ITP who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). The efficacy of eltrombopag for achieving hemostatic platelet counts (≥ 50 × 109/L) in previously treated adult ITP patients with more than 6 months' disease duration is around 80% (Wong et al., 2017). However, there is insufficient data on safety and efficacy of TPO-RAs in newly diagnosed ITP patients. AIM: The aim of this trial (NCT04346654; CETB115JDE01) is to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce a sustained response off treatment in comparison to dexamethasone monotherapy in newly diagnosed primary ITP patients. METHODS: This is a Phase II, multicenter, randomized (1:1), open-label study (see Figure 1). Arm A: Eltrombopag + short course of high-dose dexamethasoneArm B: 1-3 cycles of high-dose dexamethasone Adult patients with newly diagnosed primary ITP who have platelet counts < 30 × 109/L and require treatment will be screened, and if eligible, will be randomized to either Arm A or Arm B. The study will be conducted in the following periods: Screening Period: Patients will be screened based on the inclusion and exclusion criteria specified below Treatment / Tapering Period: Arm A: Patients will receive eltrombopag in combination with a short course of high-dose dexamethasone beginning at day 1. The dose of dexamethasone will be 40 mg QD (daily; quaque die) for 4 consecutive days and limited to 1 cycle. The starting dose of eltrombopag will be 50 mg QD in order to achieve the target platelet count of ≥ 50 × 109/L. Patients who reach platelet counts ≥ 30 × 109/L and maintain counts ≥ 30 × 109/L during the tapering phase (week 20 - week 26) will be eligible for treatment discontinuation starting from week 26. During the tapering phase, eltrombopag will be decreased by 25 mg every 2 weeks to a minimum dose of 25 mg every other day for all patients. Arm B: Treatment in the control arm consists of 1-3 cycles of high-dose dexamethasone administered orally at a dose of 40 mg QD for 4 consecutive days at 4 weeks intervals. Patients will be treated up to 12 weeks during the treatment period with dexamethasone. If the platelet counts are > 150 × 109/L no further course of dexamethasone will be given. Patients who reach platelet counts ≥ 30 × 109/L and maintain counts ≥ 30 × 109/L after 1-3 cycles of dexamethasone treatment will be eligible for treatment discontinuation. Observation period: After completion of the treatment period, patients will be observed for sustained response off treatment defined as: maintain platelet counts ≥ 30 × 109/L after treatment discontinuation and no bleeding events ≥ Grade II and without the use of any rescue therapy until week 52 and week 78 respectively, after study start The study is designed to include 106 adult patients with newly diagnosed primary ITP at 25 sites in Germany. Patients meeting any of the following criteria are not eligible for inclusion in this study: Previous history of treatment for ITPPatients with diagnosis of secondary thrombocytopeniaPatients who have life threatening bleeding complications per physician´s discretionPatients with a history of thromboembolic events or known risk factors for thromboembolism The primary objective is the rate of sustained responses off treatment at 52 weeks. Key secondary objectives include the duration of sustained response off treatment, the rate of sustained response off treatment at 78 weeks as well as patient-oriented outcomes for health-related quality of life. Currently, the study is recruiting patients, expected to be completed by 2021. CONCLUSION: This trial will evaluate the potential of eltrombopag in combination with steroids to increase the rate of sustained response off treatment in comparison to steroids alone in patients with previously untreated primary ITP. Disclosures Matzdorff: Novartis Oncology:Consultancy, Other: Honoraria paid to institution;Amgen GmbH:Consultancy, Other: Honoraria paid to institution;Grifols Deutschland GmbH:Consultancy, Other: Honoraria paid to institution;Swedish Orphan Biovitrium GmbH:Consultancy, Other: Honoraria paid to institution;UCB Biopharma SRL:Consultancy, Other: Honoraria paid to institution;Roche Pharma AG:Other: Family stockownership.Binder:DGHO:Honoraria, Other: Speaker Activity;Amgen GmbH:Honoraria, Other: Speaker Activity;Deutsche Krebsgesellschaft:Honoraria, Other: Speaker Activity;Merck Serono GmbH:Honoraria, Other: Speaker Activity;OSHO:Membership on an entity's Board of Directors or advisory committees;Medconcept GmbH:Honoraria, Other: Speaker Activity;Tumorzentrum Anhalt:Honoraria, Other: Speaker Activity;Janssen-Cilag GmbH:Honoraria, Other: Speaker Activity;DFG:Research Funding;Art tempi:Honoraria, Other;Sanofi-Aventis Deutschland:Honoraria, Other;Uniklinikum Hamburg:Honoraria, Other;event lab GmbH:Honoraria, Other: Speaker Activity.Nimmerjahn:Novartis Pharma GmbH:Honoraria.Meyer:Amgen GmbH:Honoraria;Novartis Pharma GmbH:Honoraria;Grifols Germany:Consultancy, Honoraria.Rummel:Roche:Honoraria;Janssen:Honoraria;Novartis Pharma GmbH:Honoraria;Celgene:Consultancy, Honoraria;Amgen GmbH:Honoraria.Tesanovic:Novartis Pharma GmbH:Current Employment.Sauer:Novartis Pharma GmbH:Current Employment. OffLabel Disclosure: Eltrombopag is an oral, small-molecule, non-peptide thrombopoietin receptor agonist (TPO-RA) that increases hematopoiesis by inducing proliferation and differentiation of early bone marrow progenitor cells leading to increased platelet production (Erickson-Miller et al 2010, Sun et al 2012). Eltrombopag has demonstrated efficacy in adult and pediatric patients with ITP and has a well-established safety profile. It is approved in Europe for the treatment of thrombocytopenia, from 6 months following diagnosis, in patients with primary ITP who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).

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Secondary AML After a History of Myelodysplastic Syndrome Is An Independent Adverse Prognostic Factor for Achievement of Complete Remission, Relapse-Free and Overall Survival. An Analysis of 2,819 Adult Patients with Newly Diagnosed AML Enrolled On Seven AMLSG Treatment Trials.

Blood ◽

10.1182/blood.v114.22.2621.2621 ◽

2009 ◽

Vol 114 (22) ◽

pp. 2621-2621

Author(s):

Sabine Kayser ◽

Konstanze Doehner ◽

Juergen Krauter ◽

Heinz A. Horst ◽

Marie von Lilienfeld-Toal ◽

...

Keyword(s):

Prognostic Factor ◽

De Novo ◽

Adult Patients ◽

Prior History ◽

Newly Diagnosed ◽

Adverse Prognostic Factor ◽

Time Period ◽

History Of ◽

Multivariable Analyses ◽

De Novo Aml

Abstract Abstract 2621 Poster Board II-597 Background: AML patients with a prior history of a myelodysplastic syndrome (MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN) (in the following referred to as secondary AML [s-AML]) in the new WHO classification are listed in the category “AML with myelodysplasia-related changes”. Patients with s-AML are considered to have an inferior outcome compared with de novo AML. Even after accounting for clinical and cytogenetic risk factors, s-AML is believed to remain an important predictor of poor outcome although mechanisms for this effect remain elusive. The minimal time period between diagnosis of MDS and occurrence of s-AML has been defined differently, 3 months by Cheson et al. (J Clin Oncol 1990) versus 6 months according to the WHO 2001 classification. Aims: To study the clinical impact of s-AML in a large cohort of patients with newly diagnosed AML, in the context of clinical characteristics as well as cytogenetics and mutational status of the NPM1, CEBPA and FLT3 genes. Methods: The study included 3,139 adult patients (median age, 53.5 years; range, 16-85 years) with newly diagnosed AML entered on 7 protocols of the German-Austrian AML Study Group (AMLSG) between 1993 and 2008. In all protocols intensive induction and consolidation therapy was used. Information on type of AML, karyotype and molecular marker status of NPM1 and FLT3 (internal tandem duplication [ITD]) was available in 2,858 of 3,139 (91%) and 2,126 of 3,139 (68%) patients, respectively. Since this report focuses on the comparison of s-AML versus de novo AML, patients with therapy-related AML (t-AML) and those lacking information on type of AML were excluded (n=200, n=120, respectively). Results: 151 of the 2,819 patients (5.4%) were classified as s-AML, 122 with at least a 6-month (s-AML-6), and 29 with a 3- to 6-month (s-AML-3) time period of a preceding MDS or MDS/MPN. Compared with patients with de novo AML, those with s-AML-6 and s-AML-3 were significantly older (62 and 58 years vs 53 years; p<0.0001), and they had significantly lower median white blood counts (WBC) (6.9 and 4.6 vs 12.5 ×109/L; p=0.005), lower percentage of bone marrow blast (50 and 52 vs 75%; p<0.0001) and of peripheral blast counts (22 and 20 vs 55%; p=0.004). Compared to patients with de novo AML, those with s-AML-6 or s-AML-3 showed more frequently MDS-related cytogenetic changes (as defined by WHO 2008) (22% and 31% vs 16%, p=0.02), whereas the frequency of cytogenetically normal AML was equally distributed (48% and 52% vs 49%). However, the distribution of molecularly defined subgroups in cytogenetically normal AML was significantly different (p<0.0001) with a lower frequency of the subgroups CEBPAmut, NPM1mut/FLT3-ITDneg and FLT3-ITDpos in s-AML-6 and s-AML-3 and a higher frequency of the triple-negative genotype (75% and 45% vs 28%). In univariable analyses for the clinical endpoints achievement of complete remission (CR), relapse-free (RFS) and overall survival (OS), s-AML-6 and s-AML-3 showed a significant inferior outcome (p<0.0001, equivalent for all endpoints). Therefore s-AML-6 and s-AML-3 were combined as s-AML for further multivariable analyses. In multivariable analyses, s-AML was consistently an independent adverse prognostic factor for the endpoints achievement of CR (OR, 0.48, p=0.0001), RFS (HR, 1.38, p=0.0005) and OS (HR, 1.27, p=0.02). In cytogenetic subgroup analyses, s-AML was an unfavorable factor for OS particularly in patients with cytogenetically normal karyotype AML (p=0.01) and in patients with the molecularly defined genotype NPM1neg, FLT3-ITDneg as well as CEBPAneg (p=0.04). In as treated analysis, younger adult patients (age <61 years) with s-AML had a beneficial effect from allogeneic hematopoietic stem cell transplantation (HSCT) on OS (p=0.008). Conclusions: In this large cohort of adult patients with newly diagnosed AML, s-AML showed consistent features irrespective of duration of prior history of MDS (>6 months versus 3 to 6 months) and was an adverse prognostic factor for achievement of CR, RFS and OS. Younger adult patients with s-AML appear to benefit from allogeneic HSCT. Disclosures: No relevant conflicts of interest to declare.

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CMET-13. THE COMPOSITION OF NEWLY-DIAGNOSED BRAIN METASTASES VARIES WIDELY BY PATIENT DEMOGRAPHICS

Neuro-Oncology ◽

10.1093/neuonc/noz175.214 ◽

2019 ◽

Vol 21 (Supplement_6) ◽

pp. vi53-vi54

Author(s):

Vasileios Kavouridis ◽

Matthew Torre ◽

Maya Harary ◽

Timothy Smith ◽

Bryan Iorgulescu

Keyword(s):

Brain Metastases ◽

Cancer Registries ◽

Pacific Islanders ◽

Prior History ◽

Newly Diagnosed ◽

Patient Demographics ◽

Asian Pacific Islanders ◽

Intracranial Metastases ◽

History Of ◽

Asian Pacific

Abstract INTRODUCTION Metastases comprise a significant subset of all intracranial neoplasms, but their national composition remains unclear and challenging to track in national cancer registries. METHODS The patient demographics and histologic distribution of newly-diagnosed brain metastasis (BM) patients aged >18yo without a prior history of cancer (2010–2015) were evaluated using the National Cancer Database, which comprises >70% of all newly-diagnosed cancers in the U.S. RESULTS 91,686 adults presented with a newly-diagnosed BM between 2010–2015. The most common sites of brain metastases overall were lung (82% of metastatic cases), breast (4.1%), melanoma (3.2%), kidney (2.9%), and colorectal (1.8%). The overall 1-year and 5-year OS rates for all metastatic tumors were 27.0% (95%CI=26.7–27.3]) and 5.3% (95%CI=5.1–5.5), respectively. The distribution of primary sites for newly diagnosed intracranial metastases varied by sex, age, and race. Compared to males, more females had BMs from breast (8.4% versus 0.8%) and fewer had BMs from kidney (1.9% versus 3.8%), melanoma (1.9% versus 4.5%), and esophagus (0.3% versus 2.0%). In young adults, particularly those 20-29yo, BMs were more likely from melanoma, genitourinary (in males), and soft tissue than adults in middle and advanced age. Lung carcinomas comprised fewer BMs in Hispanics (66%) compared to Whites (82%), Blacks (83%), and Asian/Pacific Islanders (85%). BMs from kidney and genitourinary primaries were higher in Hispanics (7.3% and 2.4% of BMs, respectively) than in Whites (2.8% and 0.3%, respectively), Blacks (1.8% and 0.1%, respectively), and Asian/Pacific Islanders (2.6% and 0.2%, respectively). Melanoma was more frequent in Whites (3.8% of BMs) and Hispanics (2.5%) compared to Blacks (0.3%) and Asian/Pacific Islanders (0.6%). CONCLUSION Our results illustrate the national distribution of newly-diagnosed BMs and investigates how the distribution varies by patient demographics.

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