Essential Thrombocythaemia in Young Adults: Clinical Characteristics and Outcomes – a Single Center Experience

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 5060-5060
Author(s):  
Grace Kam ◽  
Richard Yiu ◽  
Ai Leen Ang ◽  
Yvonne SM Loh ◽  
Yeh Ching Linn ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Disease Progression ◽  
Young Patients ◽  
Medical Attention ◽  
Thrombotic Risk ◽  
Risk Of Death ◽  
Increased Risk ◽  
Thrombosis Rate

Abstract Abstract 5060 Less than 20% of patients with essential thrombocythemia (ET) are diagnosed below the age of 60. Patients with ET have increased risk of thrombosis and bleeding and potential for progression to myelofibrosis (MF) or acute myeloid leukaemia (AML). In limited studies of young patients, the clinical course has been relatively benign with low rates of transformation to AML or MF. Thrombohemorrhagic events are generally few, but higher than that of the general population. This study aims to characterize of a group ET patients diagnosed at age ≤40, their thrombotic and hemorrhagic events, disease progression and treatment given. Patients were identified through a single institution MPN registry. This is an IRB approved registry that captures comprehensive information about patients with ET. Data on patient demographics, treatment, and disease-related events were obtained. Patients were diagnosed from 1975–2011, using either WHO or PVSG criteria depending on date of diagnosis. Kaplan-Meier method was used for survival analysis. 59 patients were diagnosed with ET at age ≤40. Median age of diagnosis was 31. 5years (range 16–40), with a median follow up of 7. 7years (0. 4–33. 8). All were of Asian descent: 81. 4% Chinese, 11. 9% Malay, 3. 4% Indian and 3. 4% Filipino. 40. 7% were male. JAK2 V617F mutation was screened for in 61%. Of these patients, 11 were positive, 25 negative for the mutation. Mean presenting counts were: WBC 10. 7 × 109/L (5. 9–21. 3), Hb 13. 6g/dL (9. 7–16. 4), platelets 957 × 109/L (449–2377). Splenomegaly was noted in 3 patients. 20. 3% had underlying hypertension, 16. 9% hyperlipidemia and 5. 1% diabetes mellitus. One patient had a prior stroke. Another had prior portal vein thrombosis. At diagnosis, 23. 7% were symptomatic, with microvascular symptoms of headache (11. 9%) and giddiness (6. 8%) being most common. The remainder were diagnosed incidentally, on health screening or when seeking medical attention for unrelated conditions. One patient presented with a myocardial infarction at diagnosis, while another had a significant bleeding post hemorrhoidectomy with drop in Hb by >2g/dL (platelet 2457 × 109/L). Based on a history of prior thrombosis, 3 patients were defined as high risk for thrombotic events. 67. 8% of patients had cytoreduction, indications being platelets ≥1500 × 109/L (n=16), presence of risk factors for atherosclerotic disease (n=11) and history/onset of thrombosis (n=5). In 8, the reason for cytoreduction could not be ascertained. Hydroxyurea was most commonly used (62. 7%), followed by anagrelide in 52. 5% and interferon 25. 4%. 5. 1% received busulphan, and 1. 7% 32P. Use of antiplatelet therapy was noted in 83. 8%, most frequently aspirin (76. 5%) and ticlopidine (11. 9%). On follow up, 2 arterial thromboses occurred (stroke, TIA), giving a thrombosis rate of 0. 39%/patients/year. Neither was a recurrent thrombosis. No venous thrombosis or major bleeds occurred. 20. 4% had minor mucocutaneous bleeding; 5 had platelets ≥1500 × 109/L at that time. 3. 4% had disease progression due to MF and another 3. 4% had AML. 3. 4% of patients died due to AML. Median survival was 33. 8years (95% confidence interval 30. 3–35. 5). Initial blood counts, presence of JAK2 and high risk disease status did not correlate with thrombotic risk, risk of death or disease progression. Use of antiplatelet agents and a platelet count ≥1500 × 109/L did not correlate with bleeding risk. Few studies have looked exclusively at young patients with ET. In this group, most patients were asymptomatic and well, ET being diagnosed incidentally. They were predominantly at low risk for thrombosis and other ET-related complications. The period of follow up was comparable to that of other studies and during that time, the rate of complications and risk of disease progression was low. The thrombosis rate of 0. 39% per patient year was less than that reported by other groups (2. 2–2. 6 thromboses/100patients/year) (Leukaemia 2007;21:1218–1223, Clin Appl Thrombosis/Hemostasis 2000;6(1):31–35) but similar to the 0. 74%/patient year reported by Barbui (Blood. Epub. June 13 2012). Overall findings generally complemented those reported by other groups. No risk factors were found to influence the occurrence of complications, but the number of events was small. Follow up of this group of patients over time is essential to see if their disease course remains benign or if complications will increase with time. Soli Deo Gloria Disclosures: Kam: Shire Pharmaceuticals: Consultancy, grant to support the MPN registry Other.

COVID-19 and hematologic diseases: Risk factors and long-term follow-up of CHRONOS19 Registry.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18715-e18715
Author(s):  
Kristina Zakurdaeva ◽  
Olga A. Gavrilina ◽  
Anastasia N. Vasileva ◽  
Sergei Dubov ◽  
Vitaly S. Dubov ◽  
...  
Keyword(s):  
Risk Factors ◽  
Mechanical Ventilation ◽  
Disease Progression ◽  
Primary Endpoint ◽  
Acute Leukemias ◽  
Risk Of Death ◽  
All Cause Mortality ◽  
Long Term Follow Up

e18715 Background: Pts with hem diseases are at high risk of COVID-19 severe course and mortality. Emerging data on risk factors and outcomes in this patient population is of great value for developing strategies of medical care. Methods: CHRONOS19 is an ongoing nationwide observational cohort study of adult (≥18 y) pts with hem disease (both malignant and non-malignant) and lab-confirmed or suspected (clinical symptoms and/or CT) COVID-19. Primary objective was to evaluate treatment outcomes. Primary endpoint was 30-day all-cause mortality. Long-term follow-up was performed at 90 and 180 days. Data from 14 centers was collected on a web platform and managed in a deidentified manner. Results: As of data cutoff on January 27, 2021, 575 pts were included in the registry, 486 of them eligible for primary endpoint assessment, n(%): M/F 243(50%)/243(50%), median age 56 [18-90], malignant disease in 452(93%) pts, induction phase/R/R/remission 160(33%)/120(25%)/206(42%). MTA in 93(19%) pts, 158(33%) were transfusion dependent, comorbidities in 278(57%) pts. Complications in 335(69%) pts: pneumonia (67%), CRS (8%), ARDS (7%), sepsis (6%). One-third of pts had severe COVID-19, 25% were admitted to ICU, 20% required mechanical ventilation. All-cause mortality at 30 days – 17%; 80% due to COVID-19 complications. At 90 days, there were 14 new deaths: 6 (43%) due to hem disease progression. Risk factors significantly associated with OS are listed in Tab 1. In multivariate analysis – ICU+mechanical ventilation, HR, 53.3 (29.1-97.8). Acute leukemias were associated with higher risk of death, HR, 2.40 (1.28-4.51), less aggressive diseases (CML, CLL, MM, non-malignant) – with lower risk of death, HR, 0.54 (0.37-0.80). No association between time of COVID-19 diagnosis (Apr-Aug vs. Sep-Jan) and risk of death. COVID-19 affected treatment of hem disease in 65% of pts, 58% experienced treatment delay for a median of 4[1-10] weeks. Relapse rate on Day 30 and 90 – 4%, disease progression on Day 90 detected in 13(7%) pts; 180-day data was not mature at the time of analysis. Several cases of COVID-19 re-infection were described. Conclusions: Thirty-day all-cause mortality in pts with hem disease was higher than in general population with COVID-19. Longer-term follow-up (180 days) for hem disease outcomes and OS will be presented. [Table: see text]

Anthracycline-Induced Clinical Heart Failure in a Cohort of 607 Children: Long-Term Follow-Up Study

2001 ◽  
Vol 19 (1) ◽  
pp. 191-196 ◽  
Author(s):  
L. C.M. Kremer ◽  
E. C. van Dalen ◽  
M. Offringa ◽  
J. Ottenkamp ◽  
P. A. Voûte
Keyword(s):  
Risk Factors ◽  
Heart Failure ◽  
Cumulative Dose ◽  
Cumulative Incidence ◽  
Clinical Status ◽  
Young Patients ◽  
Increased Risk ◽  
Patients At Risk ◽  
Clinical Heart Failure

PURPOSE: To determine the early and late cumulative incidence of anthracycline-induced clinical heart failure (A-CHF) after anthracycline therapy in childhood and to identify associated risk factors. PATIENTS AND METHODS: The cumulative incidence of A-CHF and the risk factors of A-CHF were assessed in a cohort of 607 children who had been treated with anthracyclines between 1976 and 1996. For 96% of the cohort, we obtained the clinical status up to at least January 1997. The mean follow-up time was 6.3 years. RESULTS: The cumulative incidence of A-CHF was 2.8%, after a mean follow-up time of 6.3 years and a mean cumulative dose of anthracyclines of 301 mg/m2. A cumulative dose of anthracycline higher than 300 mg/m2 was associated with an increased risk of A-CHF (relative risk, 11.8; 95% confidence interval, 1.6 to 59.5) compared with a cumulative dose lower than 300 mg/m2. The estimated risk of A-CHF increased with time after the start of anthracycline chemotherapy to 2% after 2 years and 5% after 15 years. CONCLUSION: Up to 5% of patients will develop A-CHF 15 years after treatment, and patients treated with a cumulative dose of anthracyclines higher than 300 mg/m2 are at highest risk for A-CHF. This is thus a considerable and serious problem among these young patients. The findings reinforce the need for strategies for early detection of patients at risk for A-CHF and for the evaluation of other chemotherapeutic possibilities or cardioprotective agents in relation to the survival.

Obstetric antiphospholipid syndrome: still a challenge

Lupus ◽  
2010 ◽  
Vol 19 (4) ◽  
pp. 457-459 ◽  
Author(s):  
RA Levy ◽  
GRR Jesús ◽  
NR Jesús
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Antiphospholipid Syndrome ◽  
Fetal Death ◽  
Obstetric Complications ◽  
Premature Delivery ◽  
Thrombotic Risk ◽  
Recurrent Abortions ◽  
Obstetric Antiphospholipid Syndrome

Obstetric complications such as fetal death, premature delivery, preeclampsia and recurrent abortions (since chromosomal or anatomic defects have been excluded) are characteristic manifestations of antiphospholipid syndrome (APS). They can occur in patients with known APS with previous arterial or venous events in any tissue or organ, or be its first and only manifestation. Pregnancy in a patient with APS is considered high risk and the full prenatal clinical follow-up must be carried with this in mind, eliminating or minimizing concomitant thrombotic risk factors.

scholarly journals Incidence of Unprovoked Venous Thromboembolic Events in Patients with Chronic Lymphocytic Leukemia (CLL)

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 1110-1110
Author(s):  
Lauren Angela Katkish ◽  
Sravanti Rangaraju ◽  
Thomas S Rector ◽  
Gerhard J Johnson ◽  
Mark A Klein ◽  
...  
Keyword(s):  
Risk Factors ◽  
Chronic Lymphocytic Leukemia ◽  
General Population ◽  
Incidence Rate ◽  
Lymphocytic Leukemia ◽  
Thrombotic Risk ◽  
Long Term Study ◽  
Increased Risk ◽  
Venous Thromboembolic Events

Abstract While lymphoma is a known risk factor for venous thromboembolism (VTE), the thrombogenicity of chronic lymphocytic leukemia (CLL) has not been investigated. One study reported a ten-fold increased risk in VTE compared to the general population, but this included both provoked and unprovoked events, making it difficult to discern the independent contribution of CLL to the thrombogenic predisposition (Whittle A et al. Leuk Res 2011; 35:419-21). Since we found no studies examining only unprovoked VTE in CLL, we sought to estimate the risk of spontaneous VTE occurring in patients with CLL in the absence of known thrombogenic risk factors. The study was approved by the institutional human subjects committee. We examined patients diagnosed with CLL between 1997-2014 by the persistent presence of ≥ 5,000 circulating small, mature, monoclonal, CD5+, CD23+ B lymphocytes (per diagnostic criteria in: Halleck M et al. Blood 2008;111:5446-56) and listed in the Minneapolis VA Tumor Registry. Clinical and laboratory data were obtained from the electronic medical record. Each patient's chart was reviewed individually for VTE, either pulmonary embolism (PE) or deep vein thrombosis (DVT), and the report of a confirmatory imaging study was examined to confirm the diagnosis. We followed patients (n = 308, 99% males, mean age 70 years) starting at the date of CLL diagnosis or the date after CLL diagnosis when the patient began follow-up at the VA and ending at last patient contact or death (122/308 [39.6%] patients died during the observation period). Each patient made at least annual contact with the VA between the start and end dates, with a mean VA follow-up time of 4.6 +/- 2.9 years; 90% patients were followed continuously at the VA since after the diagnosis of CLL. Patients were excluded if there was a preceding period of immobility, serious illness requiring hospitalization, diagnosed hypercoagulable state, or recent chemotherapy known to be associated with increased thrombotic risk. The total follow-up time was 1408 patient-years, during which 10 unprovoked VTE occurred, for an estimated incidence rate of unprovoked VTE of 0.71 per 100 patient-years, 95% CI [0.38, 1.32]. Eight VTE were originally diagnosed at our institution. Confirmatory imaging was available for the 2 VTE diagnosed elsewhere. All 10 patients were males between the ages of 55 to 95 years at the time of CLL diagnosis. Six had DVT, three had PE, and one had both DVT and bilateral PE. Nine patients had symptoms that prompted diagnostic imaging, while one PE was diagnosed incidentally on a chest CT scan. Nine patients had bulky lymphadenopathy at the time of VTE, but these did not compress the relevant vein in any patient. Small, stable monoclonal paraproteinemia was identified in 2/5 VTE patients tested (one patient at 0.39 and 0.35 g/dL, one patient at 0.32 and 0.27 g/dL). At time of development of VTE, 4, 1, 1 and 3 patients had Rai stage I, II, III or IV CLL, respectively. Thus, while there were relatively few events, there did not appear to be any clear relationship of VTE with advanced CLL stage. Recurrence of unprovoked VTE occurred in only 1 of 10 (10%) patients. In a study of the general population in our region of the US (Olmsted County, MN), the overall incidence of VTE in 70-74 year old males was 0.65 per 100 patient-years (Silverstein M et al. Arch Internal Med 1998;158:585-93). Since 17-47% of all VTEs were unprovoked in various population series (Cushman M et al. Thromb Haemost 2001;86[suppl 1]:OC2349, Heit JA et al. Arch Intern Med 2002;162:1245-8, Spencer FA et al. J Thromb Thrombolysis 2009;28:401-9, White RH Circulation 2003;107:I-4-8), the incidence rate of unprovoked VTEs observed in CLL patients in the current series (0.71; 95% CI: 0.38, 1.32) approximates the expected incidence of unprovoked VTEs in the general population. Further, the development of unprovoked VTE would have been expected to be skewed towards patients with advanced stage (Rai stages III/IV), and/or have been associated with more frequent recurrences, if CLL was an independently thrombogenic condition. Conclusion: This is the first long-term study of unprovoked VTE in patients with CLL, with >1400 patient-years of follow-up. The low incidence of unprovoked VTEs observed in our study suggests that primary thromboprophylaxis may not be required in patients with CLL who do not have additional risk factors for thromboembolism. Disclosures No relevant conflicts of interest to declare.

scholarly journals Risk of a Diagnosis of Dementia for Elderly Medicare Beneficiaries after Intensive Care

2015 ◽  
Vol 123 (5) ◽  
pp. 1105-1112 ◽  
Author(s):  
Carmen Guerra ◽  
May Hua ◽  
Hannah Wunsch
Keyword(s):  
Risk Factors ◽  
Intensive Care ◽  
Critical Illness ◽  
General Population ◽  
Hospital Discharge ◽  
Medicare Beneficiaries ◽  
Risk Of Death ◽  
Increased Risk ◽  
Diagnosis Of Dementia

Abstract Background Critical illness is likely associated with an increased risk of dementia, but the magnitude remains uncertain. Methods The cohort was a random 2.5% sample of Medicare beneficiaries who received intensive care in 2005 and survived to hospital discharge. Patients were matched with general population controls (age, sex, and race) with 3 yr of follow-up. The authors used an extended Cox model to assess the risk of a diagnosis of dementia, adjusting for the known risk factors for dementia, and the competing risk of death. Results Among 10,348 intensive care patients who survived to hospital discharge, dementia was newly diagnosed in 1,648 (15.0%) over the 3 yr of follow-up versus 12.2% in controls (incidence per 1,000 person-years, 73.6; 95% CI, 70.0 to 77.1 vs. 45.8; 95% CI, 43.2 to 48.3; hazard ratio [HR], 1.61; 95% CI, 1.50 to 1.74; P < 0.001). After accounting for the known risk factors in the year before the index hospitalization, the risk of receiving a diagnosis of dementia remained increased in patients who received intensive care (adjusted HR, 1.43; 95% CI, 1.32 to 1.54; P < 0.001). Inclusion of identifiable risk factors accrued during the quarter of critical illness accounted for almost all of the increased risks (adjusted HR, 1.09; 95% CI, 1.00 to 1.20; P = 0.06). Conclusions Elderly critical care survivors have a 60% increased relative risk, but only 3% increased absolute risk, of receiving a diagnosis of dementia in the subsequent 3 yr compared with the general population. This increased risk is not accounted for by risk factors preexisting the critical illness. Surveillance bias, which increases the likelihood of receiving a diagnosis of dementia, could account for some or all of these additional risks.

scholarly journals 2613. The Epidemiology of Respiratory Syncytial Virus (RSV) in People with Immune Dysfunction Seen at a Tertiary Hospital Between 2010 and 2017

2019 ◽  
Vol 6 (Supplement_2) ◽  
pp. S908-S909
Author(s):  
Qiuju Li ◽  
Joanne Reekie ◽  
Marie Helleberg ◽  
Henrik Sengeløv ◽  
Søren Schwartz Sørensen ◽  
...  
Keyword(s):  
Risk Factors ◽  
Respiratory Syncytial Virus ◽  
Immune Dysfunction ◽  
Solid Organ ◽  
Cell Transplant ◽  
Risk Of Death ◽  
Increased Risk ◽  
Syncytial Virus ◽  
The Impact

Abstract Background Persons with a compromised immune system are at increased risk of complications related to respiratory syncytial virus (RSV) but the risks are not well defined. We aimed to investigate the prevalence of RSV infection, associated risk factors and complications in a large population of people with immune dysfunction. Methods Persons with immune dysfunction, first seen at Copenhagen University Hospital, Rigshospitalet, between January 1, 2010 and February 21, 2017, aged ≥18 were included. RSV testing and positivity (positive PCR or antigen test) was determined through the Danish Microbiology Database. Generalized estimating equations logistic regression was used to investigate the risk factors for RSV positivity; Cox regression was used to assess the impact of RSV positivity (time-updated) on mortality in the first 12 months after first visit. Results The study included 42,567 persons, of which 3,356 (7.9%, 95% CI 7.6%-8.1%) were tested for RSV at least once during follow-up, with 2,374 (71%) tested in the first 12 months. Stem cell transplant (HSCT) and solid-organ transplant (SOT) recipients had the highest proportion of persons tested for RSV (66.0%, 95% CI 62.9%–69.1% and 31.6%, 95% CI 29.0%–34.2%, respectively). Of those tested, 256 (7.6%, 95% CI 6.7%–8.5%) had ≥1 positive RSV test (figure). After adjustment, HSCT and SOT recipients, as well as other hematologic and rheumatologic patient groups were more likely to have a positive RSV test compared with persons seen in the infectious disease department. Fifty-seven RSV-related complications were identified in 53/256 (20.7%, 95% CI 15.7%-25.7%) persons positive for RSV (table), of which 24 (45.3%) were HSCT recipients and 18 (34.0%) were other hematologic patients. In the 12 months after first department visit, 9,451 (22%) patients died; persons with RSV had an increased risk of short-term mortality (aHR 1.77, 95% CI 1.19–2.64), adjusting for sex, age, patient group and flu positivity. Conclusion Patients with a hematological or rheumatological condition and SOT recipients had the highest odds of contracting RSV, with hematological patients in particular at an excess risk of RSV-related complications. RSV was associated with an increased risk of death in the first 12 months of patient follow-up. Disclosures All authors: No reported disclosures.

Incidence of Second Malignant Neoplasms (SMN) after Haematopoetic Stem Cell Transplant.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 551-551
Author(s):  
Samar Kulkarni ◽  
Michael Potter ◽  
Mark Ethell ◽  
Radovan Saso ◽  
Jennifer Treleaven ◽  
...  
Keyword(s):  
Risk Factors ◽  
Stem Cell ◽  
High Risk ◽  
Brain Tumours ◽  
Primary Diagnosis ◽  
Advanced Stage ◽  
Haematological Malignancies ◽  
Stage Disease ◽  
Increased Risk

Abstract From August 1973 to December 2003, 1651 patients (median age: 28yr., range: 1–74 yr.; M: 982, F: 669) received stem cell transplants for haematological malignancies (Ac. Leuk: 1246, Chr. Leuk: 182, other: 223). Donor was allogeneic (n=1088), autologous (n=537) or syngeneic (n=26) and conditioning was with (n=1243) or without TBI (n=408). Primary disease was classed as high risk in 714 and low risk in 937 patients. Dose of TBI was ≤9.5 Gy (n=342), 10.5 Gy (n=613) or ≥ 10.5 Gy (n=282) delivered in single fraction (n=1015) or multiple fractions (n=222). Donor was sibling (n=900), matched unrelated (n=135) or mismatched family member (n=53). Cranial top-up radiation was used in 357 patients. Source of stem cell was marrow (n=1312), PBSC (n=320) or both (n=19). GVH prophylaxis was CyA alone (n=527), CyA with other agent (n=516) or other measures (n=45). Of all the patients 1515 received single transplant but 136 received more than one transplant. Data was analysed as of 01/07/2006. Of all the patients 897 (54.3%) survived for at least 1 yr. post transplant and the median follow-up in this group was 27yr. Second cancers developed in 50 cases with the incidence of 2.6% at 5yr (95% CI: 1.3–3.9), 7.5% at 10 yr (95% CI: 4.9–10.1) and 11.5% at 15yr (95%CI: 7.6–15.4). Site of second malignancy was brain (n=10), breast (n=6), cervix (n=3), GIT (n=2), lung (n=1), skin (n=9), sarcoma (n=3), thyroid (n=1), oral cavity (n=4), MDS (n=6), CML (n=1), ovary (n=1) and non EBV related lymphoma (n=3). In univariate analysis 10 yr. probability of developing SMN was significantly higher with chronic GVHD (5% vs. 10%, p=0.008, high risk disease (5% vs. 11%, p=0.0007), cranial RT (4% vs. 18%, p=0.015) and ALL as primary diagnosis (4% vs. 10%, p=0.06). In multi-variate analysis advanced stage disease (RR: 2.4, 95% CI: 1.3–4.2, p=0.004), Chr. GVHD (RR: 2.5, 95% CI: 1.4–4.4, p=0.003) and cranial RT (RR: 2.3, 95% CI: 1.2–4.4, p=0.01) were independently associated with increased risk of SMN. There was strong interdependence between cranial RT and ALL as primary diagnosis. It was noted that the multi-variate risk factors were different for site specific SMNs. For brain tumours cranial RT (RR: 12.8, 95% CI:2–75.5, p=0.006) and age <16 yr at the time of transplant (RR:15.7, 95% CI:1.9–130.6, p=0.011) were associated with increased risk. For epithelial malignancies Chr. GVHD (RR: 5.8, 95% CI: 2.2–15.5, p=0.0005), female sex (RR: 3.0, 95% CI:1.1–8.3, p=0.033) and advanced stage disease (RR: 3.6, 95% CI: 1.4–9.4, p=0.01) were associated with increased risk. No independent risk factors could be identified for development of skin or haematological malignancies. 25 patients have died due to SMN (49%) at a median of 31 months. Survival was significantly better in skin SMNs (85%) as compared to haematological SMNs (20%), brain tumours (30%) and epithelial SMNs (56%)(p=0.02). This single centre analysis shows that the risk of developing SMN does not relate to the type of conditioning and increases with longer follow-up. The risk factors vary for the different sites of SMNs. These patients need long term cancer screening.

Application of the International Prognostic Score of Thrombosis for Essential Thrombocytemia(ET) (IPSET-Thrombosis) in a Cohort of ET Patients: Experience from Gruppo Laziale for Myeloproliferative Ph Negative Neoplasms

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2821-2821
Author(s):  
Michele Cedrone ◽  
Barbara Anaclerico ◽  
Francesca Paoloni ◽  
Roberto Latagliata ◽  
Marco Montanaro ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Risk Group ◽  
Prognostic Score ◽  
High Risk Group ◽  
Thrombotic Risk ◽  
International Prognostic Score ◽  
Essential Thrombocytemia ◽  
Observation Period

Abstract INTRODUCTION: Essential Thrombocytemia (ET) is the most common of the myeloproliferative neoplasms, vascular complications contribute mostly to both morbidity and mortality. The ability to identify thrombotic risk of the individual patient is necessary for a correct therapeutic management. Traditionally, risk stratification for thrombosis in ET pts was based on the respective absence and/or presence of either age >60 years or history of thrombosis. Recently, the IPSET score (International Prognostic Score Of Thrombosis for ET) was developed to better predict the occurrence of thrombotic events in ET patients. Risk factors included in the new score were: age, cardiovascular risk factors, previous thrombosis, presence of JAK 2 V617F mutation. AIM: to evaluate the validity of IPSET-thrombosis score in a cohort of ET patients from "Gruppo Laziale for Myeloproliferative Ph negative Neoplasms" in predicting thrombosis incidence. METHODS: from January 1978 to December 2011 we observed 1249 ET patients, median follow up was 105 months (range 12.1-417.7). We were able to retrospectively evaluate all the IPSET risk factors in 680 ET patients and estimated the clinical implication of the IPSET-thrombosis score system. According to the score 27.3 %, 19.1% and 53.5% of pts were stratified in low, intermediate and high risk group respectively. RESULTS: median age at the time of diagnosis was 61.8 yrs (range 19.9 -94; 64% females), median hemoglobin was 14 g/dl (range 6-20); median leukocyte count was 8.8 x 109/L (range 1,2-57); median platelets count was 812x 109/L (range 108-3582). We observed, during a median follow-up of 200 months, 82 thrombotic events (total incidence 17,89 %). According IPSET-thrombosis risk, in our ET population was documented a statistically different thrombosis free survival (TFS) (Gray test 0.1316): 85%, 78%, 77% in low,intermediate and high risk group respectively. During all the observation period the intermediate and high risk group showed a similar probability of thrombotic events. CONCLUSIONS: in our retrospective study the IPSET score was able to differentiate the rate of thrombosis events in low-risk (0-1 risk factors) from that of intermediate (>= 2 risk factors) and high risk (>= 3 risk factors) ET pts. Unfortunately the intermediate and high risk groups show a similar incidence of thrombotic events during the whole observation period. We were not able to verify the clinical benefit resulting from the different treatment strategies. Because treatment options in ET pts are tailored according to thrombotic risk, and since we have specified therapeutic indications for patients with low and high risk, it is necessary to validate the score IPSET in a large prospective, long term study to discriminate a true "intermediate" subset of patients for which there are no currently defined therapeutic guidelines. Disclosures No relevant conflicts of interest to declare.

Abstract 4565: Optimal Timing Of Stage-2 Palliation For Hypoplastic Left Heart Syndrome

Circulation ◽  
2008 ◽  
Vol 118 (suppl_18) ◽  
Author(s):  
Edward J Hickey ◽  
Christopher A Caldarone ◽  
Eugene H Blackstone ◽  
Thomas Yeh ◽  
Christian Pizarro ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Survival Models ◽  
Optimal Timing ◽  
Risk Category ◽  
Cumulative Hazard ◽  
Risk Of Death ◽  
Increased Risk ◽  
Stage 1 ◽  
High Risk Children

Background Because optimal timing of stage-2 palliation of HLHS is contentious, we explored associations between length of time between stage-1 and stage-2 (“interstage interval”) and survival. Methods A multi-institutional cohort of neonates (N=703;1994 –2001) who underwent Norwood palliation for HLHS was studied. Survival models were constructed for each of stage-1 and stage-2 and their combined cumulative hazard was defined. Results A constant, persisting hazard for death was prominent ≈2 months after Norwood (“interstage death”). Risk factors for interstage death included: smaller weight at diagnosis, longer DHCA duration, smaller indexed tricuspid annulus, mitral stenosis, older age at Norwood, smaller distal arch dimension and sternal splinting (all P <.01, >50% bootstrap reliability). Following stage-2 palliation (N=404, median interval 5.8 months after Norwood), 32 died before subsequent Fontan completion. Risk factors for these deaths included: earlier stage-2 ( P <.0001, 60% reliability), younger age at Norwood and smaller RV dimensions at diagnosis. Performing stage-2 within 2 months of Norwood conferred exponential increased risk of death. Cumulative hazard exposure (figure ) was minimized with interstage interval ≈ 3 months. For low risk patients intervals >4 months were optimal. For high-risk children survival was optimized with ≈ 2 month interstage interval. Conclusions Optimal timing of stage-2 palliation is dependent on patients’ risk category. High-risk children may have improved survival by early progression to stage-2; no such benefits are seen for lower risk children. The interstage interval will not be safely reduced below 2 months.

scholarly journals New risk model is able to identify patients with a low risk of progression in systemic sclerosis

RMD Open ◽  
2021 ◽  
Vol 7 (2) ◽  
pp. e001524
Author(s):  
Nina Marijn van Leeuwen ◽  
Marc Maurits ◽  
Sophie Liem ◽  
Jacopo Ciaffi ◽  
Nina Ajmone Marsan ◽  
...  
Keyword(s):  
Machine Learning ◽  
Systemic Sclerosis ◽  
High Risk ◽  
Disease Progression ◽  
Risk Model ◽  
Immunosuppressive Drugs ◽  
Low Risk ◽  
Expert Assessment ◽  
Risk Of Progression

ObjectivesTo develop a prediction model to guide annual assessment of systemic sclerosis (SSc) patients tailored in accordance to disease activity.MethodsA machine learning approach was used to develop a model that can identify patients without disease progression. SSc patients included in the prospective Leiden SSc cohort and fulfilling the ACR/EULAR 2013 criteria were included. Disease progression was defined as progression in ≥1 organ system, and/or start of immunosuppression or death. Using elastic-net-regularisation, and including 90 independent clinical variables (100% complete), we trained the model on 75% and validated it on 25% of the patients, optimising on negative predictive value (NPV) to minimise the likelihood of missing progression. Probability cutoffs were identified for low and high risk for disease progression by expert assessment.ResultsOf the 492 SSc patients (follow-up range: 2–10 years), disease progression during follow-up was observed in 52% (median time 4.9 years). Performance of the model in the test set showed an AUC-ROC of 0.66. Probability score cutoffs were defined: low risk for disease progression (<0.197, NPV:1.0; 29% of patients), intermediate risk (0.197–0.223, NPV:0.82; 27%) and high risk (>0.223, NPV:0.78; 44%). The relevant variables for the model were: previous use of cyclophosphamide or corticosteroids, start with immunosuppressive drugs, previous gastrointestinal progression, previous cardiovascular event, pulmonary arterial hypertension, modified Rodnan Skin Score, creatine kinase and diffusing capacity for carbon monoxide.ConclusionOur machine-learning-assisted model for progression enabled us to classify 29% of SSc patients as ‘low risk’. In this group, annual assessment programmes could be less extensive than indicated by international guidelines.

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