scholarly journals Practice Patterns and Real-Life Outcomes for Patients with Acute Promyelocytic Leukemia in the United States

2021 ◽  
Author(s):  
Jan Philipp Bewersdorf ◽  
Stephanie Prozora ◽  
Nikolai A. Podoltsev ◽  
Rory Michael Shallis ◽  
Scott F Huntington ◽  
...  
Keyword(s):  
Acute Promyelocytic Leukemia ◽  
Adverse Outcome ◽  
Real Life ◽  
The United States ◽  
Population Based ◽  
Promyelocytic Leukemia ◽  
Adverse Outcomes ◽  
Hospital Death ◽  
Newly Diagnosed ◽  
Logistic Regression Models

Acute promyelocytic leukemia (APL) is associated with a favorable long-term prognosis if appropriate treatment is initiated promptly. Outcomes in clinical trials and population-based registries vary; potential explanations include a delay in treatment and lower adherence to guideline-recommended therapy in real-world practice. We used the Vizient Clinical Data Base (CDB) to describe demographics, baseline clinical characteristics, and treatment patterns in newly diagnosed APL patients during the study period of April 2017 - March 2020. Baseline white blood cell count (WBC) was used to assign risk status and assess treatment concordance with National Comprehensive Cancer Network guidelines. Logistic regression models examined adjusted associations between patient, hospital, disease characteristics, and adverse outcomes (in-hospital death or discharge to hospice). Among 1,464 APL patients, 205 (14.0%) experienced an adverse outcome. A substantial subset (20.6%) of patients did not receive guideline-concordant regimens. Odds of adverse outcomes increased with failure to receive guideline-concordant treatment (OR: 2.31 [95% CI: 1.43 - 3.75]; p=0.001), high-risk disease (OR: 2.48 [1.53 - 4.00]; p<0.001) and increasing age (≥60 years: OR: 11.13 [95% CI: 4.55 - 27.22]; p<0.001). Higher hospital AML patient volume was associated with lower odds of adverse outcome (OR: 0.44 [0.20 - 0.99] for ≤ 50 vs. >200 AML patients/year; p=0.046). In conclusion, in this large database analysis, 14.0% of newly diagnosed APL patients died or were discharged to hospice. A substantial proportion of patients did not receive guideline-concordant therapy, potentially contributing to adverse outcomes.

Incidence and Clinical Features of Acute Promyelocytic Leukemia In a Population-Based Canadian Cohort

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4374-4374 ◽  
Author(s):  
Kristjan Paulson ◽  
David Szwajcer ◽  
Arshad Ahsanuddin ◽  
Pascal Lambert ◽  
Donna Turner ◽  
...  
Keyword(s):  
Acute Promyelocytic Leukemia ◽  
Fusion Gene ◽  
Clinical Care ◽  
Epidemiological Data ◽  
The United States ◽  
Population Based ◽  
Promyelocytic Leukemia ◽  
High Rate ◽  
Cns Disease ◽  
Rare Malignancy

Abstract Abstract 4374 Introduction: Acute promyelocytic leukemia (APL) is a rare malignancy whose causes and epidemiology are not well known. The incidence of APL in the United States was estimated to be 0.22 cases per 100,000 population, and a higher incidence has been suggested in specific geographic or ethnic sub-groups. However, clinical and epidemiological data on APL arise primarily from clinical trials and single centre experiences rather than population-based data, which may lead to selection bias. We studied a cohort of APL cases in a well defined population using the diagnostic gold standard of polymerase chain reaction (PCR) testing. Methods: The Canadian province of Manitoba, with a population of 1.2 million, has a single referral centre for the definitive diagnosis and clinical care of all acute leukemia patients. Only one laboratory is capable of performing PCR based testing for the PML-RARA fusion gene product. We examined consecutively diagnosed patients with APL based on PCR testing from January 1999 until July 31 2010 in order to determine the incidence, clinical characteristics, and outcomes of patients with APL. Results: Over the 11.5 year period of observation, 24 patients were diagnosed with APL. Based on the 2006 Canadian census, the annual incidence of APL in Manitoba is thus 0.18/100,000 population. These cases did not appear to be distributed evenly, with an average of 1.1 cases diagnosed annually between 2002 and 2008 compared to six cases diagnosed in the most recent period between 2009 and 2010 (relative to 2002–2008, incident rate ratio (IRR) 2.61, p = 0.075) and ten cases diagnosed between 1999 and 2001 (IRR 2.94, p = 0.023). Symptomatic CNS disease at diagnosis was present in 3 (12.5%) patients. Thirteen patients developed differentiation syndrome during the course of their therapy (54.1%). In all but one patient, ATRA was able to be successfully resumed. Ten patients presented with DIC (41.7%). Conclusions: In this population-based study, we noted episodic clustering of new cases and a relatively high proportion of CNS disease at diagnosis. While these observations may be due to normal fluctuations in the patterns a rare disease, this could also suggest an underlying environmental trigger. Additional investigation is required to confirm or refute this hypothesis, due to the relatively small numbers in this cohort. The high rate of CNS disease suggests that this finding maybe more common than previously thought. As the pathogenesis of APL remains unclear, these observations deserve additional epidemiological investigation in larger cohorts. Disclosures: Kumar: Celgene: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Acute promyelocytic leukemia: A population-based study on incidence and survival in the United States, 1975-2008

Cancer ◽  
2012 ◽  
Vol 118 (23) ◽  
pp. 5811-5818 ◽  
Author(s):  
Yiming Chen ◽  
Hagop Kantarjian ◽  
Haijun Wang ◽  
Jorge Cortes ◽  
Farhad Ravandi
Keyword(s):  
United States ◽  
Acute Promyelocytic Leukemia ◽  
The United States ◽  
Population Based ◽  
Promyelocytic Leukemia ◽  
Population Based Study

A Population Based Study of 408 Cases of Acute Promyelocytic Leukemia Showing Changes in Epidemiology and Prognosis During Two Decades: Impact of Oral Arsenic Trioxide,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3597-3597
Author(s):  
Wing-Yan Au ◽  
Dennis Ip ◽  
Oscar Mang ◽  
Kit Fai Wong ◽  
Margaret Ng ◽  
...  
Keyword(s):  
Hong Kong ◽  
Arsenic Trioxide ◽  
Acute Promyelocytic Leukemia ◽  
Early Death ◽  
The United States ◽  
Population Based ◽  
Promyelocytic Leukemia ◽  
Male Gender ◽  
Older Age

Abstract Abstract 3597 Background. The incidence of acute promyelocytic leukemia (APL) shows racial variations. Population based epidemiologic studies are feasible because of common diagnostic criteria and treatment. The prognosis of APL has substantially improved since the advent of all trans-retinoic acid (ATRA) and arsenic trioxide (As2O3). Material and methods. Data on survival and relapse of consecutive APL patients in Hong Kong from 1991 to 2011 were obtained from the Hong Kong Cancer Registry (with at least 98% reporting and complete follow-up), and verified by hospital records. Data were censored at the end of July 2011. Potential factors impacting on survival including age, platelet count (Plat), white blood cell count (WBC), gender; 5-year cohort and As2O3 maintenance were analyzed by logistic regression. Results. Four hundred and eight cases of APL (198 men, 210 women) at a median age of 41 (3–89) years were registered. There was a rise in cases number with successive 5-year cohorts, but the WHO standardized age incidence rate (WSIR) was unchanged (Table 1). At diagnosis (Dx), the median hemoglobin was 8.4 (2.9–14.9) g/dL, WBC 17.7 (0.3–250) × 109/L (>10 × 109/L in 129 cases) and Plat 35 (3–270) × 109/L (< 40 × 109/L in 282 cases). Early death (within 30 days of Dx) occurred in 88 cases. Complete remission (CR) was achieved in 318 cases. Outcome was unknown in 2 cases. The incidence of early death decreased progressively with each 5-year cohort (p=0.035), but was positively correlated with older age (p<0.001), high WBC (p<0.001) and male gender (trend only, p=0.06). From CR1, the median follow-up was 83 (0–249) months. Relapse occurred in 108 cases. The 5-year relapse rate fell from 54% (no maintenance) to 16% (p<0.001) with the adoption of ATRA (n=110) and oral As2O3 (n=88; since 2001) maintenance. Relapse rates were lower in patients receiving As2O3 than ATRA maintenance (17% versus 38%; p=0.008). Risk factors predicting relapse were older age (p=0.001), no oral-As2O3 maintenance (p=0.003), high WBC (p=0.023) and male gender (trend only, p=0.056). For relapsed patients, 5-year overall survival (OS) from CR1 was improved from 67% with ATRA (1993–1998) to 96% with oral-As2O3 (since 1999–2011) treatment. The causes of deaths after CR1 were APL relapse (n=35), second cancer (n=8), bone marrow transplantation (BMT, n=7), chemotherapy (n=3) and unrelated causes (n=6). Allogeneic BMT was not performed since 1999. For the last 5-year cohort (2006–2011), there were no APL related deaths after CR1. With reduction of induction death, and improvement in treatment and prevention of relapses, the 5-year OS from diagnosis increased from 44% for the first 5-year cohort to 80% for the last 5-year cohort. On multivariate analysis, age (p<0.001) and cohort period (p=0.031) were determinants of OS. Conclusions. Population based incidence data showed that APL in Hong Kong was more prevalent than the United States (0.15–0.18/100000/year). Oral-As2O3 has markedly changed the outcome of APL patients. Early death is now the greatest problem curtailing survival of APL patients. Disclosures: No relevant conflicts of interest to declare.

Maternal and Infant Adverse Outcomes Associated with Mild and Severe Preeclampsia during the First Year after Delivery in the United States

2019 ◽  
Vol 37 (04) ◽  
pp. 398-408
Author(s):  
Thanh G.N. Ton ◽  
Mihoko V. Bennett ◽  
Devin Incerti ◽  
Desi Peneva ◽  
Maurice Druzin ◽  
...  
Keyword(s):  
United States ◽  
Adverse Outcome ◽  
The United States ◽  
Population Based ◽  
Adverse Outcomes ◽  
Severe Preeclampsia ◽  
First Year ◽  
Inverse Association ◽  
Mild Preeclampsia ◽  
Collider Bias

Objective The burden of preeclampsia severity on the health of mothers and infants during the first year after delivery is unclear, given the lack of population-based longitudinal studies in the United States. Study design We assessed maternal and infant adverse outcomes during the first year after delivery using population-based hospital discharge information merged with vital statistics and birth certificates of 2,021,013 linked maternal–infant births in California. We calculated sampling weights using the National Center for Health Statistics data to adjust for observed differences in maternal characteristics between California and the rest of the United States. Separately, we estimated the association between preeclampsia and gestational age and examined collider bias in models of preeclampsia and maternal and infant adverse outcomes. Results Compared with women without preeclampsia, women with mild and severe preeclampsia delivered 0.66 weeks (95% confidence interval [CI]: 0.64, 0.68) and 2.74 weeks (95% CI: 2.72, 2.77) earlier, respectively. Mild preeclampsia was associated with an increased risk of having any maternal adverse outcome (relative risk [RR] = 1.95; 95% CI: 1.93, 1.97), as was severe preeclampsia (RR = 2.80; 95% CI: 2.78, 2.82). The risk of an infant adverse outcome was increased for severe preeclampsia (RR = 2.15; 95% CI: 2.14, 2.17) but only marginally for mild preeclampsia (RR = 0.99; 95% CI: 0.98, 1). Collider bias produced an inverse association for mild preeclampsia and attenuated the association for severe preeclampsia in models for any infant adverse outcome. Conclusion Using multiple datasets, we estimated that severe preeclampsia is associated with a higher risk of maternal and infant adverse outcomes compared with mild preeclampsia, including an earlier preterm delivery.

scholarly journals Real-life outcomes of unselected acute promyelocytic leukemia patients: a single-center 14-year experience

2020 ◽  
Vol 58 (3) ◽  
pp. 138-145
Author(s):  
Omer Faruk Akcay ◽  
Haci Hasan Yeter ◽  
Yahya Buyukasik
Keyword(s):  
Acute Promyelocytic Leukemia ◽  
Survival Rates ◽  
Real Life ◽  
Disease Free Survival ◽  
Early Death ◽  
Population Based ◽  
Promyelocytic Leukemia ◽  
Term Survival ◽  
Probability Of Survival ◽  
All Trans Retinoic Acid

AbstractBackground. After the inclusion of all-trans retinoic acid (ATRA) into the treatment of Acute Promyelocytic leukemia (APL), a notable improvement concerning the survival rates of patients with APL has been observed. However, the population-based studies demonstrated that there was no marked improvement in the survival of patients after the 2000s. We aim to describe the clinical response and prognosis of adult patients diagnosed with APL and examine the change in these outcomes by the time period of diagnosis.Methods. We retrospectively reviewed thirty-six unselected APL patients who were diagnosed between September 2003 and February 2016.Results. The probability of survival at two years was 58%, while disease-free survival (DFS) was 87%. The overall early death (ED) rate was 33% and remain stable over time [42% in 2003–2009 vs. 24% in 2010–2016 (p=.20)]. In addition, the 2-year overall survival (OS) rates were 47% in 2003–2009 and 70% in 2010–2016 (p=.29), and no differences were noted. Univariate analyses showed possible predictors of poor OS were defined as leukocytosis (≥10x109/L), high Sanz score, hemorrhage, infection, disseminated intravascular coagulopathy (DIC) at presentation and microgranular morphologic subtype.Conclusions. This study shows that long-term survival remains low in APL patients, particularly related to a high ED rate. Initiatives to reduce ED are exceedingly substantial for improving the survival in APL.

scholarly journals Real Life Experience with ATRA-Arsenic Trioxide Based Regimen in Acute Promyelocytic Leukemia - Updated Results of the Prospective German Intergroup Napoleon Registry

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2815-2815
Author(s):  
Uwe Platzbecker ◽  
Eva Lengfelder ◽  
Katharina S Goetze ◽  
Christoph Röllig ◽  
Michael Kramer ◽  
...  
Keyword(s):  
Arsenic Trioxide ◽  
Acute Promyelocytic Leukemia ◽  
Research Funding ◽  
Real Life ◽  
Study Groups ◽  
Promyelocytic Leukemia ◽  
Intermediate Risk ◽  
Newly Diagnosed ◽  
Front Line ◽  
Line Therapy

Abstract Background: Standard therapy of acute promyelocytic leukemia has long relied on the combination of All-trans-retinoic acid (ATRA) and chemotherapy. The introduction of arsenic trioxide (ATO) in APL treatment has allowed achievement of similarly high remission and survival rates coupled with significantly reduced myelosuppression. Recent results of the APL0406 trial by the GIMEMA-AMLSG-SAL study groups showed that the combination of ATRA and arsenic trioxide (ATO) is superior to standard ATRA and chemotherapy (CHT) in front-line therapy of low/intermediate risk acute promyelocytic leukemia (APL). The implications of these results for the clinical practice of APL patients in Germany have been uncertain given the fact that ATO is not formally licensed for front-line therapy of APL. Aim:In order to provide evidence and a reflection of the clinical reality of APL patient care in Germany an intergroup APL registry (National acute promyelocytic leukemia (APL) observational study, NAPOLEON) was recently initiated by several AML study groups. Methods:Eligible patients are adults at least 18 years of age with newly diagnosed or relapsed APL not beyond the first year of diagnosis. Here we report the first analysis on the series of patients prospectively enrolled into this registry. The study was conducted in accordance with the Declaration of Helsinki, received IRB approval by all participating centers and was registered at ClinicalTrials.gov (NCT02192619). Results: As of August 1st 2016, 88 patients have been included into the study with a median age of 57 years (range 22-87). All had newly diagnosed APL (100%) with 66% (n=58) being of low/intermediate risk according to the Sanz score. Out of those patients 76% (n=44) received an ATO-ATRA based induction regimen followed by a median of 4 courses of consolidation (according to the APL 0406 study).Of 41 patients evaluable for response to induction, 40/41 (98%) patients achieved complete remission (CR) with the ATRA-ATO arms. Early death rate within 30 days of therapy was 2% (1/44). After a median follow-up of 12 months, the event-free survival, cumulative incidence of relapse and overall survival at 12 months for these patients were 97%, 0% and 97%, respectively. Therapy was well tolerated and no new safety signals have been obtained. Conclusion:These real life data from a prospective German registry provide further evidence for the safety and sustained anti-leukemic efficacy of ATRA-ATO in low/intermediate risk APL. These results further support ATRA-ATO as the new standard of care in this clinical setting. Table Demographic, clinical and laboratory characteristics of the eligible patients. Table. Demographic, clinical and laboratory characteristics of the eligible patients. Disclosures Platzbecker: TEVA: Honoraria, Research Funding. Greiner:BMS: Research Funding. Thiede:AgenDix: Employment, Other: Ownership. Hochhaus:BMS: Honoraria, Research Funding; Novartis: Honoraria, Research Funding; Pfizer: Honoraria, Research Funding; ARIAD: Honoraria, Research Funding.

scholarly journals Remission in Patients with Acute Promyelocytic Leukemia Treated with Arsenic Trioxide (varitrinox) As the First Line in Colombia

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-3
Author(s):  
Gustavo Milone ◽  
Samuel Sarmiento Doncel ◽  
Carol Agudelo Rico ◽  
Fabiola Vizcarra Reyes ◽  
Gina Alejandra Diaz Mosquera ◽  
...  
Keyword(s):  
High Risk ◽  
Arsenic Trioxide ◽  
Acute Promyelocytic Leukemia ◽  
Conflicts Of Interest ◽  
Promyelocytic Leukemia ◽  
Remission Induction ◽  
Intermediate Risk ◽  
Newly Diagnosed ◽  
First Line ◽  
Risk Patients

Acute promyelocytic leukemia (APL) is a subtype of Acute Myeloid Leukemia (AML) in which a chromosomal translocation t (15; 17) (q22; q12) is generated by fusing produces a hybrid PML / RARα gene, generating an altered signal . The combination of transretinoic acid (ATRA) plus arsenic trioxide (ATO) has been shown to be superior to ATRA plus chemotherapy in the treatment of newly diagnosed standard risk patients with acute promyelocytic leukemia (APL) in several countries. The objective of the present study is to describe the frequency of remission in patients with acute promyelocytic leukemia who were administered as a first line Arsenic Trioxide (varitrinox) during the period from November 2017 to June 2020 in Colombian patients. Methods: Retrospective observational and descriptive study of 12 patients diagnosed with acute promyelocytic leukemia treated with ATO Arsenic trioxide (Varitrinox) as first line, the source of information was provided by the treating hematologists (medical records) by filling out the technical concept format. Active pharmacovigilance scientist in Colombia, this format keeps the identification information of the patient anonymized and the confidentiality of the data is guaranteed as well as compliance with the rules of good clinical practice. Results: Twelve patients with age range between 22 and 69 years with a median age of 34.0 were analyzed. It was found in the analysis that 100% had induction hematologic remission with a median of 45 days. 75% of patients received ATO + ATRA and were at low and intermediate risk, the remaining 25% received ATRA + ATO + Chemotherapy and were at high risk, and intermediate risk. 91.7% of molecular remission in consolidation was obtained and it was measured in cycle 3 by means of PCR (undetectable), 8.3% (n = 1) was positive 3% and is finishing consolidation. Regarding the most frequent adverse events, intravascular coagulation (n = 9), neutropenia (n = 6) and thrombocytopenia (n = 6) were observed. 75% of patients are disease-free, 16.7% are on maintenance (they received ATO + ATRA + Induction chemotherapy) and 8.3% are on consolidation. So far, none of the patients under study have died. Conclusions: Our results support the use of ATO (Varitrinox) in newly diagnosed APL patients (as first line), as a care strategy for low, intermediate and high risk patients. The role of ATRA-ATO is guaranteed in other studies where they manage patients of different risks. Key words: Arsenic trioxide, leukemia promyelocytic acute, leukemia myeloid acute, remission induction, tretinoin. Disclosures No relevant conflicts of interest to declare.

Counterpoint: Implementing Population Genetic Screening for Lynch Syndrome Among Newly Diagnosed Colorectal Cancer Patients—Will the Ends Justify the Means?

2010 ◽  
Vol 8 (5) ◽  
pp. 606-611 ◽  
Author(s):  
Michael J. Hall
Keyword(s):  
Lynch Syndrome ◽  
Screening Program ◽  
Real Life ◽  
The United States ◽  
Third Party ◽  
Clinical Expertise ◽  
Newly Diagnosed ◽  
Dna Testing ◽  
Limited Effectiveness ◽  
Clinical Resource

Inherited mutations in 1 of 4 known mismatch repair genes (MLH1, MSH2, MSH6, PMS2) are associated with various cancer risks collectively referred to as Lynch syndrome. Roughly 3 of every 100 new colorectal cancers (CRCs) have an underlying Lynch mutation. Tumor-based screening for Lynch among all patients with newly diagnosed CRC could theoretically improve the ability to identify Lynch and prevent cancer among at-risk family members, but the patient-level and social implications of this approach must be carefully considered before adopting this strategy. Poorly addressed issues include the role/timing of informed consent for testing, access and cost barriers associated with genetic counseling and DNA testing, psychosocial burdens to the thousands of middle-aged and elderly patients with CRC coping with surgical and chemotherapy treatments and poor prognosis, the need for providers to warn third-party relatives of risk for Lynch syndrome, limited effectiveness of screening, and the cost burden to society when poor DNA testing uptake, test limitations, and modest screening compliance are considered. Diverse barriers to the success of a population-based Lynch screening program in the United States remain (e.g., clinical resource needs, financial limitations, clinical expertise gaps, educational deficits). Data supporting clinical efficacy (feasibility) and effectiveness (real-life performance) are critical before important policy changes are adopted, especially where issues of hereditary cancer risk and genetic privacy are involved.

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