Introduction: Up to 20% of patients (pts) with essential thrombocythemia (ET) and up to 10-15% of pts with polycythemia vera (PV) are diagnosed before the age of 40 years and the percentage is even ...lower in pts with primary myelofibrosis (PMF). Pregnancy (Pcy) in MPN is associated with an increased incidence of thrombotic and bleeding events and obstetric complications with live birth rates of 50% to 70%. There are no evidence-based guidelines for the management of these pts and most of the data come from case series of few pts. There are no validated variables that can predict outcomes in pregnant women with MPN.
Objetives:
Primary: To learn the incidence of thromboembolic, hemorrhagic and OC in pregnant women with MPN in Argentina.
Secondary: To evaluate parameters such as mutational status, platelet count, history of complications in previous Pcy, use of aspirin (ASA), low molecular weight heparin (LMWH) or interferon (IFN) and their relationship with obstetric outcomes.
Materials and methods: Retrospective evaluation of medical records of pts with diagnosis of MPN and Pcy. Quantitative variables were expressed as median and interquartile range (IQR) and qualitative variables as total number and percentage (%). Fisher's exact test was used to analyze variables and their association with events and Wilcoxon test for platelet counts.
Results: A total of 30 Pcy in 23 women with a diagnosis of MPN were recorded, 20 Pcy in pts with ET, 4 Pcy in pts with PV and 6 MF pregnancies. Pcy was planned in 56.6% of cases. There were OC in 15/19 previous Pcy. The median age was 32.9 years (IC25-75% 30-36 years). Mutational status was assessed in 22 pts: 9/22 JAK2 +, 7/22 CALR +, 1/22 triple-negative, 1/22 JAK2-CALR- and MPL not performed, 6/22 JAK2- CALR and MPL without data. The overall number of live births and first trimester spontaneous abortions (SA) were 26(86%) and 4(13.3%) respectively, without cases of second (2T) and third trimester (3T) fetal loss or stillbirths. There was a case of neonatal death in a patient with MF. We detected 13 obstetric events (ET: 9/20, PV: 2/4, and MF 2/6): 4 SA, 5 cases of fetal growth retardation, 1 partial placental abruption and 3 placental hematomas. There was no significant association between JAK2 mutational state, history of complications in previous Pcy, platelet count, use of ASA, LMWH or IFN and obstetric outcomes. Two of the pts who had SA presented smoking as a cardiovascular risk factor. There was heterogeneity among hematologist regarding therapeutic management: ASA was indicated in 24 Pcy and LMWH in prophylactic doses during Pcy in 15. In 21/25 cases LMWH was indicated as postpartum prophylaxis, only in 3/21 it was extended for 6 weeks(wks). Before Pcy 17 cases received cytoreductive treatment: 2 with HU and 1 with anagrelide that were discontinued at the time Pcy was confirmed, 5 with peg IFN, and 9 conventional IFN. During Pcy 8 continued with cytoreductive treatment (6/9 conventional IFN, 2/9, pegIFN, including 1 patient previously receiving HU) and 5 initiated IFN (1/5 conventional, 4/5 pegIFN). Platelet counts in pts who did not require cytoreduction dropped from 600 x 109/L (290-1596 x 109/L) at the beginning of Pcy to 470 (240-1548) x 109/L, p=0.0068, and 423 (240-843) x 109/L, p=0.0005, in the 2T and 3T, respectively, with non-significant increase after delivery to 494 x 109/L (122-1600 x 109/L). No cases of maternal thrombosis and 2 episodes of major postpartum bleeding who required surgical intervention one of them with hysterectomy were found.The median time of delivery was at 38 wks of gestation (IC25-75% 36.5-38.5). Vaginal delivery was performed in 6 Pcy, caesarean section (CS) was required due to emergency in 2, because of lack of fetal progression in 2, and 16 were scheduled CS. The median birth weight was 2900g (IC25-75% 2500-3300g). No congenital malformations in live births were detected.
Conclusions: Pcy is a rare but high-risk event in MPN pts with 11/30 OC in this cohort. The live birth rate was high compared to the literature. The risk of MC was low, without thrombotic complications and 2/30 episodes of major postpartum bleeding. There was no correlation between mutational status, platelet count or treatments received and obstetric outcomes. There was a significant reduction in the number of platelets in pts with thrombocytosis without requiring cytoreduction. The high percentage of pts who finished their pregnancy with scheduled CS is highlighted.
No relevant conflicts of interest to declare.
Background: ALL in adults is considered a heterogeneous disease with poor prognosis. However, adolescents and young adults (AYA) display intermediate characteristics as compared with children. Risk ...groups and response predictors are essential to guide the treatment.
Aims: The aim of this report was to evaluate the experience of Ph-negative ALL in AYA patients (pts) treated with the pediatric Argentine type-BFM protocol, assessing predictors of response in terms of overall survival (OS) and event-free survival (EFS).
Methods: We performed a retrospective multicenter analysis of AYA pts diagnosed between 2013 to 2021, from 16 Argentine institutions who were treated following the pediatric Argentine type-BFM protocol. Response to prednisone (PR) at day(d) 8 (<1.0 and ≥1.0 x10 -9 blasts) in peripheral blood (PB) and complete remission (morphological remission -MCR- together with negative minimal residual disease -MRD- by flow cytometry) were evaluated. Events were defined as relapse, refractoriness or death. Chi2/Fisher's exact test, Kaplan-Meier / log rank test and Cox regression were used for statistical analysis.
Results: One hundred and seventy-three patients similarly distributed among private and public institutions were analyzed. The median (Md) age was 22 years (15-40) with a male predominance (66%). At diagnosis, 23% presented central nervous system (CNS) involvement, 79.5% B and 20.5% T immunophenotype, 29% CD20 positive, 16% adverse cytogenetic/molecular findings and 29% no data. Ph-like screening was not routinely performed assessed only in 13% revealing two CRLF2 and one PDGFRB rearrangements. Twenty-one (12%) of pts underwent allogeneic bone marrow transplantation (Allo-HSCT) at 1 st line, 20 with MRD negative and 1 MRD positive at the time of the procedure.Of 171 pts, 155 (91%) achieved MCR, 12 (7%) were refractory, 3 (2%) died prior/during induction and 1 (0.6%) patient dropped out of treatment; 144 (84%) achieved negative MRD, 22 (13%) positive MRD and 5 (3%) were not evaluated. The Md to reach CR and MRD (-) was 33d (IQR 31-42) and 35d (IQR 32-73), respectively. With a Md follow-up of 16 months (1-79), 52 pts (30%) relapsed and 68 pts (39%) died. Early deaths at 3 months occurred in 8 (5%) pts, 5 related to treatment and 3 to disease. The Md OS and EFS were 58 and 34 months, respectively. Prognostic predictors analyzed were sex, white blood counts (WBC) at diagnosis (limit of 30 x10 -9/L), age (limit of 30 years), CNS involvement, cytogenetic/molecular findings, PR at d8, MRD in bone marrow (BM) at d15, 33 and 78. In the univariate analysis, response at d8, MRD at d15, 33 and 78 were useful to predict both OS and EFS, adding age and WBC in terms of OS. Similar results were obtained when patients who underwent Allo-HSCT were censored at the time of the procedure. (Table). In the multivariate analysis, positive MRD at d33 and d78 (and WBC in OS) maintained their independent adverse prognostic impact in relation to OS and EFS.
Allo-HSCT showed benefits in pts with MRD >0.1% at d33 and >0.01% at d78 in terms of OS (d33: NR vs. 11 m, p =0.048, HR 0.3, 95%CI 0.1-0.9; d78: NR vs. 8 m, p =0.009, HR 0.4, 95%CI 0.2-0.8) and EFS (d33: NR vs. 6 m, p =0.022, HR 0.2, 95%CI 0.1-0.8; d78: NR vs. 5 m, p =0.003, 0.1 HR, 95%CI 0.02-0.5). The adverse risk of positive MRD at day 33 and 78 in terms of OS and EFS was overcome when pts were transplanted.
Summary/Conclusion: The results show that positive MRD at d33 and 78 independently impacted on OS and EFS in our series and that Allo-HSCT overcame their adverse risk. Our data confirms the relevance of using pediatric protocols in AYA. On the other hand, the importance of prognostic tools in order to improve the outcome of AYA patients, reinforcing their inclusion in current guidelines.
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No relevant conflicts of interest to declare.
Summary
The role of Ann Arbor staging in determining treatment intensity after achieving a negative positron emission tomography (PET) has not been established in classical Hodgkin lymphoma (cHL). ...Patients with stage I–IV cHL, received three cycles of ABVD (doxorubicin, bleomycin, vinblastine, dacarbazine) and an interim PET scan (PET3). PET3‐negative patients received no further therapy. PET3‐positive patients received three additional cycles of ABVD plus involved‐field radiation therapy or salvage chemotherapy, if refractory to ABVD, and were re‐evaluated by PET scan (PET6). Study endpoints were 3‐year progression‐free survival (PFS) and overall survival (OS) rates. Two hundred and thirty‐nine patients with early‐stage and 138 with advanced‐stage were evaluable. Overall, 260 patients (70%) were PET3‐negative and had higher 3‐year PFS (90% vs. 65%; P < 0·0001) and OS (98% vs. 92%; P = 0·007) rates than PET3‐positive patients. All PET3‐negative patients, regardless of disease stage at diagnosis, achieved similarly good PFS (90–91%; P = 0·76) and OS (97–99%). The only independent prognostic factor for PFS was PET3‐negativity (Hazard ratio 3·8; 95% confidence interval 2·4–6·3; P < 0·0001). This study suggests that cHL patients who achieve a negative PET3 following ABVD have an excellent outcome, regardless of stage at diagnosis. An appropriately powered, phase III trial will be necessary to confirm these findings.
Background
Response to TKI is the most important predictor of survival in CML and its early acquisition predicts the achievement of deep molecular responses (MR) (Baccarani et al. Blood 2013). In ...current ELN recommendations, the optimal time point to achieve Major Molecular Response (MMR) is defined at 12 months (mo) after diagnosis of CML however, not achieving MMR is not a failure criterion at any time point (Saussele et al, Blood 2014). There are controversies about when CML therapy must be changed in pts who do not achieve MMR after 12 mo of treatment. We wonder if pts achieving late MMR are less likely to achieve deep MR or are at risk of progression to accelerated phase (AP) or blast crisis (BC).
Objectives
We aim to identify predictive factors for achievement of deep MR and improvement of failure-free survival (FFS). Other objective was to determine if delay in MMR acquisition predicts outcomes in order to justify an optimal landmark for changing TKI reducing the risk of failure.
Methods
Between 2000 and 2015, 122 consecutive adult pts with CP CML were treated with various TKI modalities. Adherence (ADH) monitoring occurred in every visit and was reported in medical records, optimal ADH defined as >90% of dose completed. Cytogenetic and MR were defined according to ELN definitions and followed the procedures as described elsewhere (Cross et al. Leukemia 2012). In particular, BCR-ABL<1% corresponds to complete cytogenetic remission (CCyR) (Lauseker et al. 2014). MMR: BCR-ABL transcript <0.1% IS. Confirmed MR4.0: BCR-ABL detection ≤0.01% IS in two consecutive analyses. Failure was defined as loss of CCyR, AP or BC. Different models were performed for CCyR at 6mo, MMR at 12mo and MMR at 24mo as predictors of MR 4.0; in all cases age and Sokal were used as covariates. Failure-risk was evaluated with Cox regression including in the model: Time to MMR, ADH and Sokal as covariates.
Results
From 122 pts diagnosed with CP CML, 110 pts received 1st line imatinib (IM) 400mg daily and constitute the basis of the analysis. Median age was 48 years (14-82), 65% were males, 82% low Sokal score. With a median follow-up of 107 mo (IQR 69.5-141) 82% pts are still on initial IM, 4.5% died and 1.8% were lost to follow-up. A total of 20 pts (18%) discontinued Imatinib, 9% due to intolerance and 9% due to failure. At 15 years, FFS was 88% and OS 95%.The proportion of pts treated with IM who achieved CCyR at 6mo and MMR at 12mo was 84% and 38% respectively. Median-time to achieve MMR and MR4.0 was 2.6 and 3.6 years respectively. By logistic regression analysis CCyR at 6mo OR 5,6 (95% CI 1,6-19,00), MMR at 12mo OR 5,3 (95% CI 1,4-21,0), ADH OR 7,0 (95% CI 1,3-37,0) and not MMR at 24mo OR 2,5 (95% CI 0,9-7,2) resulted independent predictors for achieving MR4.0 and confirm earlier observations. In Cox model of proportional hazards for risk of failure, time to achieve MMR HR 1,02 (95% CI 1,01-1,04), ADH HR 6.1 (95% CI 1,38-26,8), and Sokal HR 4,7 (95% CI 1,12-19,3) were independent predictors of failure. Time to achieve MMR is a continuous variable, so the HR associated with it, is equivalent to a 2% monthly increase in the risk of failure. To calculate HR in other time intervals, the product of the coefficient model (B) and time of interest (in mo) were used as an exponent of the base of the natural logarithm (e). Not reaching MMR at 12 mo correlates with higher risk of failure: hazard-risk (HR) 1.30, (95% CI 1.28-1.30) and the risk increases with every passing year not achieving MMR (Table 1). Table 1Time to MMR as predictor of progressionNo MMR achievement in different time intervalsHRCI 95%After 12 mo of TKI1,30(1,3-1,3)After 24 mo of TKI1,70(1,7-1,7)After 30 mo of TKI2,21(2,2-2,2)After 60 mo of TKI3,74(3,7-3,8)
Conclusions
The prognostic value of CCyR at 6mo and MMR at 12mo was confirmed. The association between early MMR and deep MR was confirmed. In this long-term follow-up Latin American real-world cohort of patients ADH was also an early predictor for achievement of deep MR. Although good responses are obtained with IM, some patients still progress, this is the reason why it is necessary to identify those who will relapse so as to consider a change in treatment to another TKI at a convenient time point. Time to achieve MMR, ADH, and Sokal Risk score were independent predictors of failure. Not achievement of MMR at 12mo correlates with increased risk of failure. The risk increases annually in proportion to the time delay. Patients who are delayed in reaching MMR should be controlled closely.
Pavlovsky:Novartis: Honoraria, Speakers Bureau; Janssen: Honoraria, Speakers Bureau. Pavlovsky:Novartis - Bristol: Speakers Bureau.
Introduction: Early reduction of BCR-ABL transcript level has been associated with improved outcomes in CML treatment. Inability to achieve early molecular response(MR) at 3 months (M3>10%) is ...considered a predictor factor for unfavourable outcome. However, the kinetics of BCR-ABL transcript level reduction measured at early time points have shown to be an independent predictor of response.The aim of this analysis was to determine whether the "M3-M6" status is critical to categorize CML patients (pts) focusing in high-risk group.
Method: Molecular monitoring was performed in all pts prior treatment (M0), at months 3 (M3), 6 (M6), 12 (M12) and every 6 months thereafter, applying Q-PCR method according international recommendations. Results of BCR-ABL1 transcript level were reported on the international scale as IS-BCR-ABL %. Optimal responses: M3≤10%, M6≤1%, M12≤0,1%. Deep responses (MR4.0): ≤0,01% or undetectable/10.000 ABL copies.
Results: A total of 70 CML pts were included, median age 49 (19-82), female 39%. First line treatment: sustained branded 81% and generic 19% TKIs. Imatinib 59%, Dasatinib and Nilotinib 41%. Sokal risk score: low (L) 51%, intermediate (In) and high (H) 49%. Optimal responses at molecular milestones: 75% at M3, 72% at M6, 61% at M12 and 53% pts achieved MR4.0. Event-free survival (EFS) was evaluated according to time point M3: M3≤10% group had significantly better EFS compared with the M3>10% (96% vs 70%; P=0.028). M3-M6 status defined 4 groups of pts: M3≤10%-M6≤1%, M3≤10%-M6>1%, M3>10%-M6≤1%, M3>10%-M6>1%. Molecular response evolution by M3-M6 status is described in Table 1. EFS stratified by groups according to combined M3-M6 responses showed significant differences: 92% for group 1, 87% for group 2, 68% for group 3, 54% for group 4. (P=0.002). M6 time point was shown to be critical in 32 high-risk pts (H+In): 17 pts with M6 ≤1% showed significant differences in MR4.0 achievement compared to 15 pts with M6 >1% (82% vs 27% P=0.02). Better EFS was observed in this high-risk group under branded vs generic TKIs treatment (97% vs 54% P=0.04). Statistical differences in deep responses and MMR at M12 were observed between branded and generic TKIs independently of Sokal risk (P=0.06, P=0.02).
Conclusions: M3≤10% pts showed a favourable evolution with better EFS than M3>10% group. However not all patients with M3<10% had a similar behavior, showing lower rates of MMR at M12 and deep responses in those pts with M6>1%. In pts with M3 >10% and optimal response at M6 also showed higher MR4.0 rate. Our study supports that M6 is a crucial endpoint to predict MMR at M12 and deep responses in CML pts.Pts with M3≤10% without optimal response at M6 (>1%) had a worse evolution than those slow responders who showed M3>10% and M6≤1%.High-risk pts are still a challenge, observing better outcomes in those under branded TKIs treatment. The M3-M6 status would be a prognostic marker of responses and EFS in chronic phase CML pts treated with TKIs. Our data support the critical role of M6 response in non-optimal M3>10% pts and intermediate and high risk Sokal score. Treatment adherence is mandatory for achieving and sustaining optimal responses. This multicentric Argentine study, reinforces the importance of clinical follow-up and molecular monitoring under IS standardization at early time points. Education on early molecular monitoring with adequate resources must continue to be an objective in our region.
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Pavlovsky:Roche: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Novartis: Consultancy, Honoraria, Speakers Bureau. Moiraghi:Novartis: Speakers Bureau; Bristol: Speakers Bureau. Varela:Novartis: Speakers Bureau; Bristol: Speakers Bureau. Enrico:Novartis: Honoraria, Patents & Royalties; Bristol Myers squib: Speakers Bureau. Brodsky:International PNH Registry: Other: -; Alexion Pharma Argentina: Speakers Bureau. Pavlovsky:Novartis: Speakers Bureau; Janssen: Speakers Bureau; Bristol Myers Squib: Speakers Bureau.
•Symptom burden in myelofibrosis patients was assessed via the MPN-SAF and MPN-10.•Thrombocytopenic patients had higher individual and total symptom scores.•Severely thrombocytopenic patients had ...different clinical features but similar QOL.•MF patients with thrombocytopenia may benefit from therapy to decrease symptoms.
Myelofibrosis is a myeloproliferative neoplasm associated with progressive cytopenias and high symptom burden. MF patients with thrombocytopenia have poor prognosis but the presence of thrombocytopenia frequently precludes the use of JAK2 inhibitors. In this study, we assessed quality of life and symptom burden in 418 MF patients with (n=89) and without (n=329) thrombocytopenia using prospective data from the MPN-QOL study group database, including the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) and Total Symptom Score (MPN10). Thrombocytopenia, defined as platelet count <100×109/L (moderate 51–100×109/L; severe ≤50×109/L), was associated with anemia (76% vs. 45%, p<0.001), leukopenia (29% vs. 11%, p<0.001), and need for red blood cell transfusion (35% vs. 19%, p=0.002). Thrombocytopenic patients had more fatigue, early satiety, inactivity, dizziness, sad mood, cough, night sweats, itching, fever, and weight loss; total symptom scores were also higher (33 vs. 24, p<0.001). Patients with severe thrombocytopenia were more likely to have anemia (86% vs. 67%, p=0.04), leukopenia (40% vs. 20%, p=0.04), and transfusion requirements (51% vs. 20%, p=0.002) but few differences in symptoms when compared to patients with moderate thrombocytopenia. These results suggest that MF patients with thrombocytopenia experience greater symptomatic burden than MF patients without thrombocytopenia and may benefit from additional therapies.
Follicular lymphoma is an indolent lymphoma with potential relapses in the course of its evolution. In the last few years, new therapeutic strategies have improved patient ́s survival. However, a ...high risk subgroup progresses early with a shorter overall survival. Our primary objective is to report the inci-dence of POD24 and its impact on OS and, as a sec-ondary objective, to evaluate the association of the FLIPI 1, FLIPI 2 and PRIMA PI scores and the clin-ical-metabolic variables with the risk of a POD24 event. Three hundred and thirty-two patients were included, 46 patients (13.8%) with a POD24 event. Factors independently associated with greater prob-ability of a POD24 event were: presence of B symp-toms (OR 2.09) and elevated LDH (OR 1.27). In this study we report a significant difference in OS between POD24 vs NO POD24 (at 60 months, 63 vs 95% with p <0.001) reinforcing the POD24 impact as a negative prognostic factor. Despite being a retrospective study and the limited number of patients, we report local data not available to date. New clinical and/or biological markers will be necessary to identify the subgroup with POD24 risk, which could benefit from an adapted therapy.
El linfoma folicular es un linfoma indolente con re-caídas potenciales en el curso de su evolución. En los últimos años nuevas estrategias terapéuticas han mejorado la sobrevida de los pacientes. Sin embargo, un subgrupo de alto riesgo progresa en forma temprana con una menor sobrevida global (SG). Nuestro objetivo primario es reportar la incidencia de POD24 y su impacto en SG y, como objetivo secundario, evaluar la asociación de los índices FLIPI 1, FLIPI 2 y PRIMA PI y las variables clíni-co-metabólicas con el riesgo de evento POD24. Se incluyeron 332 pacientes, 46 pacientes (13,8%) con evento POD24. Los factores asociados en forma in-dependiente a POD24 fueron: presencia de síntomas B (OR 2.09) y LDH elevada (OR 1.27). En este estu-dio reportamos diferencia significativa en SG entre POD24 vs NO POD24 (a 60 meses, 63 vs 95% con p <0.001) reforzando el impacto de POD24 como factor pronóstico negativo. A pesar de tratarse de un estudio retrospectivo y de número limitado de pacientes, reportamos datos locales no disponibles a la fecha. Serán necesarios nuevos marcadores clínicos y/o biológicos que permitan identificar el subgrupo con riesgo POD24 y que podría beneficiarse de una terapia adaptada.
BACKGROUND:
The presence of constitutional symptoms has been associated with increased mortality risk in myelofibrosis (MF) (Blood 2010;115(9):1703-8). New therapies exist which alleviate the severe ...symptom burden profile observed in MF patients but are only approved for use in those with intermediate-2 or high risk disease (N Engl J Med 2012;366:787-798). However, it has been proposed that there are patients who may benefit from symptom based treatment regardless of prognostic score (Am Soc Hematol Educ Program 2014;2014:277-286). We have recently characterized symptom score cutoffs at which patients would statistically benefit from treatment based on symptom scores alone (Scherber et. al. EHA 2016: a2250). These treatment thresholds included aMyeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS or MPN-10) total score of greater than or equal to 20, a worst individual item score of greater than 5, or a combined criteria of those with both an MPN-10 total score of greater than or equal to 20 and a worst individual item score greater than 5. This abstract represents an additional analysis of our MF cohort to better characterize the profile of patients who meet criteria for symptom-based therapy.
METHODS:
Patient demographics, symptom burden via the MPN-10 score (JCO 2012;30(33)4098-103), and disease traits were collected from MF patients and their physicians at a single time point during therapy. Previously we identified MPN-10 cutoffs via AkaikeÕs Information Criterion (AIC) analysis (Ecology 2014;95: 631-6), which represented the optimal model among all models specified for the data at hand to determine which patients would most benefit from symptom-directed therapy.
RESULTS:
Demographics. 695 MF patients without previousruxolitinib therapy were included in this analysis. Overall, of 455 patients (65.4%) fit a cutoff of having a single worst symptom item of greater than 5/10. 401 patients (57.7%) had a MPN-10 score of equal to or greater than 20. A total of 381 (54.8%) patients fit both of these criteria. A distribution of worse MPN-10 individual scores is shown in Table 1. Mean TSS score was 26.4 (SD=17.7).
Symptom Criteria Associations. Demographics and disease traits: Neither mean age or age greater than 60 was significantly associated with meeting any of the symptom score cutoff criteria. Females were significantly more likely to meet any of the symptom score cutoffs (for all criteria, p=0.0003 or less). Patients with splenomegaly, particularly spleen size of greater than 15cm below the LCM, were significantly more likely than those with a normal sized spleen to meet any of the three criteria (spleen enlargement of any size p=0.014 or less; spleen greater than 15cm p=0.0114 or less). Patients who met any of the three symptom criteria tended to have a longer MPN duration, although this trend did not meet significance. A prior history of thrombosis was not associated with achieving any cutoff criterions.
Symptom burden: Individuals who met the any symptom criteria were significantly more likely to have higher DIPSS prognostic risk score (for all p=0.0002 or less).
Laboratory values: For those meeting criteria for a worst symptom greater than 5, mean WBC was 11.7 vs 9.1 x 109/L (p=0.025) and platelet count was 238.7 versus 329.1 (p=0.023). For those meeting criteria for a TSS greater than or equal to 20, mean WBC was 11.8 vs 9.5 x 109/L (p=0.04). For individuals meeting both criteria, mean WBC was 11.9 vs 9.5 x 109/L (p=0.034). The presence of peripheral blasts were significantly more common in patients with an individual worst symptom score greater than 5 (p=0.0364). Hemoglobin level was not significantly associated with symptom criteria for any cutoffs.
CONCLUSION:
Our analysis indicates that patients who would be treated based on symptom criteria are similar to patients who would be treated based on high risk features such as high DIPSS prognostic score, concerning blood count abnormalities (i.e., leukocytosis, thrombocytopenia, presence of peripheral blasts), and splenomegaly (particularly massive splenomegaly). Thrombosis history and age were not associated with criterion cutoff assignment, and it is notable that elderly age nor history of thrombosis alone would likely alter treatment choice other than anticoagulation. This data supports that JAK2 inhibitor treatment be strongly considered in patients meeting symptom based criteria.
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Dueck:Bayer: Honoraria. Kiladjian:Novartis: Honoraria, Research Funding; AOP Orphan: Membership on an entity's Board of Directors or advisory committees, Research Funding. Zweegman:Celgene: Honoraria, Research Funding; Janssen: Honoraria, Research Funding; Takeda: Honoraria, Research Funding. Schouten:Sanofi: Consultancy; Novartis: Consultancy. Etienne:ARIAD: Speakers Bureau; Pfizer: Speakers Bureau; novartis: Consultancy, Speakers Bureau; BMS: Speakers Bureau. Harrison:Shire: Honoraria, Speakers Bureau; Gilead: Honoraria, Speakers Bureau; Baxaltra: Consultancy, Honoraria, Speakers Bureau; Incyte Corporation: Honoraria, Speakers Bureau; Novartis: Consultancy, Honoraria, Other: travel, accommodations, expenses, Research Funding, Speakers Bureau. Radia:Novartis: Honoraria; Pfizer: Honoraria. Cervantes:Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Baxalta: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AOP Orphan: Membership on an entity's Board of Directors or advisory committees. Vannucchi:Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Mesa:Promedior: Research Funding; Celgene: Research Funding; CTI: Research Funding; Gilead: Research Funding; Incyte: Research Funding; Galena: Consultancy; Ariad: Consultancy; Novartis: Consultancy.
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