Myelodysplastic Syndromes in Patients Younger Than Age 50

2006 ◽  
Vol 24 (34) ◽  
pp. 5358-5365 ◽  
Author(s):  
Andrea Kuendgen ◽  
Corinna Strupp ◽  
Manuel Aivado ◽  
Barbara Hildebrandt ◽  
Rainer Haas ◽  
...  

Purpose Myelodysplastic syndromes (MDS) mainly occur in the elderly but can affect younger individuals too. The latter require special consideration to identify suitable candidates for allogeneic stem-cell transplantation, a potentially curative approach carrying a high risk of treatment-related complications. Patients and Methods We report the largest series of young MDS patients as yet, including 232 patients younger than 50 years. Their clinical characteristics and prognosis are compared with 2,496 patients older than 50 years. Results Survival was significantly longer in the younger versus older age group (40 v 23 months, respectively; P < .00005). The difference arose from patients belonging to the low- and intermediate-I–risk categories of the International Prognostic Scoring System (median survival not reached v 45 months, respectively; P < .00005). In contrast, survival was identical for both age groups (8 months for both younger and older patients; P = .81) in the intermediate-II–and high-risk categories. Established classification systems and risk scores were applicable to young patients with primary MDS. Interestingly, a particularly large difference in median survival time was seen between the intermediate-I–and intermediate-II–risk groups (176 v 8 months, respectively). For low-risk patients, the overall survival rate was more than 86% at 20 years. Conclusion According to these results, aggressive treatment approaches should rarely be recommended to younger MDS patients belonging to the low and intermediate-I risk groups.

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5541-5541
Author(s):  
Aref Al-Kali ◽  
Rong He ◽  
Mrinal M Patnaik ◽  
David S Viswanatha ◽  
Patricia T. Greipp ◽  
...  

Abstract Background: Myelodysplastic syndromes (MDS) are rare hematological neoplasms that are more typically seen in elderly patients. Young patients (< 50 years old) have been reported to comprise between 3-6% in the Surveillance, Epidemiology and End Results (SEER) (Ma X et al, Cancer 2007; Rollison R et al, Blood 2008) with a better overall survival (OS). We hereby report the characteristics of young MDS patients with long survival follow-up. Methods: A total of 1012 MDS patients' data from Oct 1993 to Dec 2015 at Mayo Clinic were reviewed after appropriate IRB approval was obtained. All cases had their bone marrow slides reviewed at our institution. Patients youngers than 50 years old (yMDS) were included as cohort 1, while the rest was included as control group (cohort 2). Prognostic factors were analyzed by univariate and multivariate analyses. Survival estimates were calculated using Kaplan-Meier curves and univariate and multivariate analyses was based on log-rank testing using JMP software version 10. Results: Characteristics: We identified 68 (7%) yMDS patients with a median age of 42 years (range, 18-49). Female gender was more common in yMDS (43% vs 31%, p= 0.05). Upon comparison between cohort 1 and 2, only platelets were significantly lower in yMDS (61 vs 102, p <0.0001), but not white blood cells, hemoglobin or degree of marrow fibrosis. MDS subtyping according to World Health Organization 2016 showed single lineage dysplasia in 15% vs 2%, multilineage dysplasia 24% vs 36% , ring sideroblasts 9% vs 15%, isolated del (5q) 1% vs 3%, excess blasts 44% vs 31%, and unclassifiable in 6% vs 5%. As expected, therapy related MDS (t-MDS) was more frequent in yMDS (33% vs 17%, p= 0.005). Transformation to acute myeloid leukemia (AML) was also more frequent in yMDS (28 % vs 11%, p= 0.0004). Compared to cohort 2, yMDS IPSS-R scores were very high, high, intermediate, low, and very low in 24% vs 13%, 29% vs 16%, 21% vs 20%, 19% vs 35%, and 7% vs 17%, respectively. Allogenic hematopoietic cell transplantation (HCT) was more frequent in yMDS (42% vs 4%, p< 0.0001). Survival outcome: Median OS was longer for cohort 1 vs cohort 2 but did not reach statistical significance (43 vs 21 months, p= 0.1). Median progression free survival (PFS) was shorter for cohort 1 vs cohort 2 but did not reach statistical significance (8 vs 12 months, p= 0.3). Median OS for cohort 1 based on R-IPSS was 44, 105, 40, 18, and 12 months for very low, low, intermediate, high and high risk groups, respectively (p= 0.09). Median OS was shorter in t-MDS vs de novo MDS in cohort 1 (13 vs 47 months, p = 0.04). Young patients who had transformed to AML had a worse median OS (18 vs 93 months, p=0.001). On multivariate analysis neither t-MDS nor R-IPSS had a statistically significant impact on OS. Conclusions: MDS is rarely diagnosed under the age of 50. IPSS-R was less powerful in detecting differences between its risk groups for this patient population, although more than half of the patients with yMDS had either high or very high risk. Among this cohort of yMDS patients, there was a significantly higher proportion with therapy-related myeloid neoplasms compared to older patients (one third of patients), with subsequent higher rates of transformation to AML and higher allogeneic HCT. In our study, we did not find an improved OS for yMDS patients compared to older patients. Disclosures Al-Kali: Celgene: Research Funding; Onconova Therapeutics, Inc.: Research Funding.


2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 7125-7125
Author(s):  
Rishi Jain ◽  
Ashwin Sridharan ◽  
Yiting Yu ◽  
K H Ramesh ◽  
Krishna Gundabolu ◽  
...  

7125 Background: The International Prognostic Scoring System (IPSS) and the revised IPSS (IPSS-R) are used to assess prognosis after diagnosis of myelodysplastic syndromes (MDS). They are based on cytogenetics, bone marrow (BM) blasts, and number and degree of cytopenias. This retrospective analysis examined racial disparities in the presentation and survival of MDS patients (pts) in Bronx, NY. Methods: MDS pts treated at the Einstein/Montefiore system between 1997-2011 were included. Diagnosis was confirmed by review of BM biopsy. Demographics, cytogenetics (for 135/161 pts), blood counts, and BM blasts at diagnosis were collected. The Kaplan-Meier method was used for median survival estimates. The two-sample t-test and chi-square analysis were used to compare clinical variables between groups. Results: 161 pts with MDS were identified. Mean length of follow-up was 3.66 years (yrs). There were significant differences between mean age at diagnosis between Hispanics and African-Americans (66.5 vs 72.3 yrs, p<0.05) and Hispanics and whites (66.5 vs 73.1 yrs, p<0.05). There was also significantly increased thrombocytopenia at diagnosis in Hispanics (p<0.05, when compared to non-Hispanics). Median survival decreased with higher risk among IPSS groups, however, the intermediate risk group in IPSS-R had a longer median survival (9 yrs) than all other risk groups. Conclusions: The cohort used to validate prognostic risk with IPSS and IPSS-R was primarily Caucasian. In our minority rich inner-city population, Hispanics presented with MDS earlier and with more thrombocytopenia. IPSS was a stronger predictor of survival than IPSS-R as the IPSS-R intermediate risk group had better survival than lower risk groups. Larger studies should be conducted to assess the applicability of IPSS-R in minority rich populations. [Table: see text]


Cancers ◽  
2021 ◽  
Vol 13 (3) ◽  
pp. 481
Author(s):  
Yan Jiang ◽  
Jean-Richard Eveillard ◽  
Marie-Anne Couturier ◽  
Benoit Soubise ◽  
Jian-Min Chen ◽  
...  

This study explores the hypothesis that genetic differences related to an ethnic factor may underlie differences in phenotypic expression of myelodysplastic syndrome (MDS). First, to identify clear ethnic differences, we systematically compared the epidemiology, and the clinical, biological and genetic characteristics of MDS between Asian and Western countries over the last 20 years. Asian MDS cases show a 2- to 4-fold lower incidence and a 10-year younger age of onset compared to the Western cases. A higher proportion of Western MDS patients fall into the very low- and low-risk categories while the intermediate, high and very high-risk groups are more represented in Asian MDS patients according to the Revised International Prognostic Scoring System. Next, we investigated whether differences in prognostic risk scores could find their origin in differential cytogenetic profiles. We found that 5q deletion (del(5q)) aberrations and mutations in TET2, SF3B1, SRSF2 and IDH1/2 are more frequently reported in Western MDS patients while trisomy 8, del(20q), U2AF1 and ETV6 mutations are more frequent in Asian MDS patients. Treatment approaches differ between Western and Asian countries owing to the above discrepancies, but the overall survival rate within each prognostic group is similar for Western and Asian MDS patients. Altogether, our study highlights greater risk MDS in Asians supported by their cytogenetic profile.


2021 ◽  
Author(s):  
Xiao Mu Hu ◽  
Xiao Yu Nie ◽  
Kai Lun Xu ◽  
Yin Wang ◽  
Feng Tang ◽  
...  

Abstract Purpose: Diffuse midline glioma (DMG), H3K27M mutant is a new entity that has become widely recognized. However, studies concerning DMG in adult patients remains rare. We did a retrospective study covering the largest amount of patients to date to analyze the clinicopathological characteristics of DMG in adult. Methods: We reviewed 117 cases of adult DMG, collected their clinical and imaging data along with pathological results including H3K27M. Summarized their features and the connection with overall survival in different age groups.Results: Among 117 cases, most tumors were located at the thalamus, 39 patients had H3K27M mutation, of whom 38 demonstrated down regulation of H3K27me3. The average overall survival of H3K27M-mutant gliomas was 13 months, while that of 78 H3K27M wild-type gliomas were 11.8 months. For young patients (age<35), The median survival time of the H3K27M-mutant was 20.1 months, while that of the H3K27M wild-type was 39.5 months. For older patients (age≥35), the median survival time of the H3K27M-mutant was 22.3 months, while that of the H3K27M wild-type was 17.1 months. The OS of patients who received biopsies, subtotal resections, and total resections were 15.8, 17.6, and 11.6 months respectively. Conclusion: The DMG in adults mainly occurred in the thalamus. H3K27M mutations tend to happen more frequently in young adults, and this genetic alteration results in a worse outcome only in young patients. For old patients, age and the approach of surgery are independent prognostic factors. Patients received biopsy instead of total resection had a better prognosis.


2020 ◽  
Author(s):  
Bin Wu ◽  
Yi Yao ◽  
Yi Dong ◽  
Si Qi Yang ◽  
Deng Jing Zhou ◽  
...  

Abstract Background:We aimed to investigate an immune-related long non-coding RNA (lncRNA) signature that may be exploited as a potential immunotherapy target in colon cancer. Materials and methods: Colon cancer samples from The Cancer Genome Atlas (TCGA) containing available clinical information and complete genomic mRNA expression data were used in our study. We then constructed immune-related lncRNA co-expression networks to identify the most promising immune-related lncRNAs. According to the risk score developed from screened immune-related lncRNAs, the high-risk and low-risk groups were separated on the basis of the median risk score, which served as the cutoff value. An overall survival analysis was then performed to confirm that the risk score developed from screened immune-related lncRNAs could predict colon cancer prognosis. The prediction reliability was further evaluated in the independent prognostic analysis and receiver operating characteristic curve (ROC). A principal component analysis (PCA) and gene set enrichment analysis (GSEA) were performed for functional annotation. Results: Information for a total of 514 patients was included in our study. After multiplex analysis, 12 immune-related lncRNAs were confirmed as a signature to evaluate the risk scores for each patient with cancer. Patients in the low-risk group exhibited a longer overall survival (OS) than those in the high-risk group. Additionally, the risk scores were an independent factor, and the Area Under Curve (AUC) of ROC for accuracy prediction was 0.726. Moreover, the low-risk and high-risk groups displayed different immune statuses based on principal components and gene set enrichment analysis.Conclusions: Our study suggested that the signature consisting of 12 immune-related lncRNAs can provide an accessible approach to measuring the prognosis of colon cancer and may serve as a valuable antitumor immunotherapy.


2018 ◽  
Vol 36 (1) ◽  
pp. 44-52 ◽  
Author(s):  
Eric J. Chow ◽  
Yan Chen ◽  
Melissa M. Hudson ◽  
Elizabeth A.M. Feijen ◽  
Leontien C. Kremer ◽  
...  

Purpose We aimed to predict individual risk of ischemic heart disease and stroke in 5-year survivors of childhood cancer. Patients and Methods Participants in the Childhood Cancer Survivor Study (CCSS; n = 13,060) were observed through age 50 years for the development of ischemic heart disease and stroke. Siblings (n = 4,023) established the baseline population risk. Piecewise exponential models with backward selection estimated the relationships between potential predictors and each outcome. The St Jude Lifetime Cohort Study (n = 1,842) and the Emma Children’s Hospital cohort (n = 1,362) were used to validate the CCSS models. Results Ischemic heart disease and stroke occurred in 265 and 295 CCSS participants, respectively. Risk scores based on a standard prediction model that included sex, chemotherapy, and radiotherapy (cranial, neck, and chest) exposures achieved an area under the curve and concordance statistic of 0.70 and 0.70 for ischemic heart disease and 0.63 and 0.66 for stroke, respectively. Validation cohort area under the curve and concordance statistics ranged from 0.66 to 0.67 for ischemic heart disease and 0.68 to 0.72 for stroke. Risk scores were collapsed to form statistically distinct low-, moderate-, and high-risk groups. The cumulative incidences at age 50 years among CCSS low-risk groups were < 5%, compared with approximately 20% for high-risk groups ( P < .001); cumulative incidence was only 1% for siblings ( P < .001 v low-risk survivors). Conclusion Information available to clinicians soon after completion of childhood cancer therapy can predict individual risk for subsequent ischemic heart disease and stroke with reasonable accuracy and discrimination through age 50 years. These models provide a framework on which to base future screening strategies and interventions.


Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3364-3364
Author(s):  
Mohamad Mohty ◽  
Myriam Labopin ◽  
Gerard Socié ◽  
Noel-Jean Milpied ◽  
Michel Attal ◽  
...  

Abstract Abstract 3364 Poster Board III-252 RIC allo-SCT is an attractive treatment modality for AML patients not eligible for standard MAC regimens. While comorbidities may represent an obstacle to the use of a MAC regimen irrespective of age, most centres usually use an age threshold around 50 years for the use of a RIC regimen. Since RIC regimen proved to significantly decrease early non-relapse mortality (NRM) after allo-SCT, investigators are currently tempted by the use of RIC regimens in lower age groups with the aim to decrease NRM and optimize allogeneic immunotherapy. The aim of this analysis was to compare outcomes (leukemia-free survival: LFS, NRM, and relapse incidence) between patients receiving the classical Bu-Cy MAC regimen vs. patients receiving a Flu-Bu RIC regimen prior to allo-SCT. Since the use of RIC in young patients is still limited, the analysis was restricted to patients aged >40 years, who were transplanted using an HLA identical sibling donor for AML in CR1. Between 2000 and 2008, 452 patients with AML in CR1 were treated with standard MAC Bu-Cy and 376 received a Flu-Bu RIC regimen, and reported to the EBMT Registry. Patients received either allogeneic PBSC or BM from HLA-identical sibling donors. As expected, patients receiving the RIC regimen were older (median: 56 y. vs. 47 y.; p<0.0001), but the median time from CR1 to allo-SCT was shorter (99 days vs. 111 d.; p<0.0001) in the MAC group. Both groups were comparable in terms of period (median year: 2005) of allo-SCT, and donor-recipient gender distribution. In terms of AML-related risk factors, WBC, cytogenetics risk groups (good: 4.1%; intermediate: 84.1%; and poor: 11.7%), and the FAB subtype (M1-M2: 50.1%; M3: 1.2%; M4-M5: 36.1%; M0-M6-M7: 12.6%) at diagnosis, were also comparable between both groups. With a median follow-up of 13 (range, 1-100) months, the 3 years probabilities of LFS were 54±3% and 49±3% in the MAC and RIC groups respectively (p=0.32). The absence of difference in LFS is explained by a lower relapse incidence in the MAC group (25+/-3% vs. 39±3%; p<0.0001), but a higher rate of NRM (28±3 vs. 20±3%; p=0.004). However, when comparing the outcome of patients aged from 40 to 50 y. (n=351; 43%) and those aged >=50 y., the picture was different. In the 40-50 age group, LFS was 55±3% vs. 37±8%, p=0.049; relapse: 26±3 vs. 53±8%, p<0.0001; NRM: 25±3 vs. 21±9%, p=0.29, in the MAC and RIC groups respectively. On the other hand, in the >=50 y. age group, LFS was 52±5 vs. 51±3%, p=0.75; relapse: 23±5 vs. 36±3%, p=0.014; NRM: 33±4 vs. 20±3%, p=0.001, in the MAC and RIC groups respectively. In summary, in this specific setting of AML in CR1, LFS is not statistically different when using MAC or RIC allo-SCT for patients older than 50 years. However, for younger patients (40-50 y. age group), the use of RIC is associated with a higher relapse rate. Prospective trials addressing the use of RIC in patients younger than 50 years are needed. Indeed, reducing toxicity without compromising disease control could be of significant benefit to many patients, but MAC (or “more intensive” RIC regimens as being tested prospectively at present), despite the hazard of increased toxicity, may be necessary in others. Thus, the trade-off between dose intensity, toxicity, and disease control will remain to be assessed for each individual patient. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4952-4952
Author(s):  
Muhammad Nadeem ◽  
Naomi Galili ◽  
Muhammad Mumtaz ◽  
Peter Daya ◽  
Jason Cheskis ◽  
...  

Abstract Abstract 4952 Introduction: MDS are a heterogeneous group of hematologic disorders associated with clonal evolution of abnormal erythroid, myeloid and megakaryocytic lineages. Risk of transformation to acute myeloid leukemia (AML) is determined by IPSS score. Common chromosomal aberrations include abnormalities of 5, 7, 8, 20 and Y. The present survival analysis is unique in that only one team of experts was involved in diagnosis and management of these patients over more than two decades. Material and Methods: Data of patients with abnormal karyotype either at first presentation or during the course of MDS was collected retroactively. FAB, IPSS and WHO classification were used. Dates of death were retrieved from social security death index. Statistical analyses were performed by using SPSS version 18. Results: Of 709 patients with abnormal karyotypes, 292(41%) were classified as RA (or refractory cytopenias), 80 (11%) RARS, 253(36%) RAEB, 45(6%) CMMoL and 34(5%) RAEB-t and 5 (1%) were unclassified. There were 271 (37%) females and 438 (63%) males with a median age of 67 yrs and 68 yrs respectively. The most frequent abnormalities affected chromosome 5 (279 or 40%); del5q/-5 with other changes was seen in 233 (83. 5 %) and isolated del5q/-5 in 79 (28. 3%). Chromosome 7 abnormalities were found in 181 (25. 5%) patients with 38 (21%) having isolated del7q/-7. Chromosomal 8 abnormality was seen in 174 (24. 5%) patients and 71 (41%) had isolated trisomy 8. Other frequently involved chromosomes were 20 and Y affecting 158 (22. 3%) and 55 (7. 8%) patients respectively. Complex karyotype with 3 or more chromosomal aberrations was seen in 201 (28. 3%) patients. Data on 700 patients was available for analysis when all chromosomal aberrations were considered according to various IPSS risk categories. The median survival was 73 months for low risk (74 patients), 33. 7 months for int-1 (303 patients), 13 months for int-2 (227 patients) and 11. 5 months for high risk (96 patients) (p=0. 000) groups. By cytogenetic abnormalities, the best median survival of 82 months (39/229) was seen in patients with del5q/-5 and low risk disease. Other risk groups with de5q/-5 showed 32, 11 and 9 months in int-1, int-2 and high risk disease respectively (p=<0. 05). The worst median survival was in patients with high risk disease and del7q/-7 (7. 4 months, 33/127) and in patients with complex karyotype (8 months, 55/197). Conclusion: Deletion 5q patients show the best median survival among low and int-1 risk groups. Our data show considerable improvement in median survival of high risk patients compared to the earlier reported survival (11. 5 versus 4 months) which probably reflects improvement due to the use of hypomethylating agents. This improvement in survival gains more significance when considering the fact that we have used the data of only those patients with chromosomal aberrations. Disclosures: No relevant conflicts of interest to declare.


Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4791-4791
Author(s):  
Pradnya D Patil ◽  
Lisa Rybicki ◽  
Donna Abounader ◽  
Hien K. Liu ◽  
Brian T. Hill ◽  
...  

Abstract The assessment of pre-transplant comorbidities is crucial for risk-stratification and is a tool to guide clinical decisions in hematological malignancy patients (pts) undergoing evaluation for stem cell transplantation (SCT). The HCT-CI scale is commonly used to identify high risk patients pre-transplant as it is highly predictive of non-relapse mortality (NRM), severity of graft versus host disease and survival after allogeneic SCT (Sorror et al, Blood 2005; Sorror et al, Blood 2014). However, its role in ASCT remains undefined. In a Center for International Blood and Marrow Transplant Research analysis, HCT-CI score of ≥3 was prognostic for higher NRM and overall mortality in ASCT patients (Sorror et al, Biol. Blood Marrow Transplant 2015), but other single institution studies have failed to confirm this observation in lymphoma patients (Jaglowski et al, Bone Marrow Transplant 2014; Dahi et al, Biol. Blood Marrow Transplant 2014; Hosing et al, Ann. Oncol. 2008). No study has correlated HCT-CI with psychosocial functioning in the setting of ASCT. We conducted a retrospective study of 350 patients with Hodgkin (N=70) and non-Hodgkin Lymphoma (N=280) who underwent ASCT at our institution from January 2009 to June 2015. Based on their HCT-CI score, patients were categorized into low risk (score 0, N=90), intermediate risk (score 1-2, N=123) and high risk (score ≥3, N=137). Psychosocial Assessment of Candidates for Transplantation (PACT) scale (0-4: 0 being poor candidate for procedure and 4 being excellent candidate) (Foster et al. BMT 2009) was used for pre-transplant psychosocial risk assessment and was available for 235 pts. We analyzed the impact of HCT-CI on transplant outcomes and its correlation with PACT scores. Our cohort was predominantly male (63%), and Caucasian (93%) with a median age of 55 years (range 20-78). The majority of the pts (96%) had good performance status with an ECOG of 0-1. The primary diagnosis was NHL in 80%, with mostly advanced stage disease (80%), and no B symptoms (93%). Median time from diagnosis to ASCT was 16 months with 75% of the pts having received ≤2 prior therapies. The median annual income based on zip code was $49406 (range $18753-127312). Disease status prior to transplant was CR/PR in 93% of the subjects. Patient and disease characteristics were comparable among the 3 HCT-CI risk groups. Higher HCT-CI risk category was associated with a lower median household income (p=0.012), higher LDH (p=0.004), more days of apheresis (p=0.026) and lower CD34+ dose x106/kg (0.046). In relation to PACT scores, higher HCT-CI was associated with poor mental health (p<0.001), decreased coping skills (p<0.001), unhealthy lifestyle habits/sedentary life (p<0.001), decreased compliance with medications/medical advice (p=0.014) and inadequate medical/transplant knowledge (p=0.017) and lower final PACT score (p<0.001). Median follow up was 35 months with 100 observed deaths, of which 72 were attributed to relapse. The 5 year estimated relapse rate, NRM, relapse free survival (RFS) and overall survival (OS) in our cohort were 42%, 11%, 49% and 62% respectively. On univariate analysis, there was no significant difference between high vs. low/intermediate HCT-CI scores on 30 day readmission rates (OR 1.24, p=0.61), 100 day mortality (OR 1.12, p=0.86), incidence of secondary malignancy (HR 0.41, p=0.17), relapse rate (HR 0.92, p=0.64), relapse mortality (HR 1.35, p=0.20), NRM (HR 0.86, p=0.71), OS (HR 1.2, p=0.37) or RFS (HR 0.98, p=0.92). Though not statistically significant, the intermediate risk group was noted to have higher 100 day mortality and NRM compared to the low and high risk groups. To our knowledge, this is first study to correlate pre-transplant HCT-CI with PACT scores in lymphoma pts who underwent ASCT. Higher HCT-CI was associated with lower socioeconomic status, poor mental health and coping skills, unhealthy lifestyle habits, decreased medical/transplant knowledge and compliance. HCT-CI did not predict survival in our cohort. Further studies are needed to investigate the association between psychosocial risk factors and HCT-CI and define their combined utility in pre-transplant risk assessment in ASCT patients. Table Patient characteristics and HCT-CI risk categories Table. Patient characteristics and HCT-CI risk categories Figure Impact of HCT-CI on OS in ASCT patients Figure. Impact of HCT-CI on OS in ASCT patients Disclosures No relevant conflicts of interest to declare.


Blood ◽  
2012 ◽  
Vol 119 (11) ◽  
pp. 2657-2664 ◽  
Author(s):  
Bart L. Scott ◽  
Ted A. Gooley ◽  
Mohamed L. Sorror ◽  
Andrew R. Rezvani ◽  
Michael L. Linenberger ◽  
...  

Abstract Studies by the International Working Group showed that the prognosis of myelofibrosis patients is predicted by the Dynamic International Prognostic Scoring System (DIPSS) risk categorization, which includes patient age, constitutional symptoms, hemoglobin, leukocyte count, and circulating blasts. We evaluated the prognostic usefulness of the DIPSS in 170 patients with myelofibrosis, 12 to 78 years of age (median, 51.5 years of age), who received hematopoietic cell transplantation (HCT) between 1990 and 2009 from related (n = 86) or unrelated donors (n = 84). By DIPSS, 21 patients had low-risk disease, 48 had intermediate-1, 50 had intermediate-2, and 51 had high-risk disease. Five-year incidence of relapse, relapse-free survival, overall survival, and nonrelapse mortality for all patients were 10%, 57%, 57%, and 34%, respectively. Among patients with DIPSS high-risk disease, the hazard ratio for post-HCT mortality was 4.11 (95% CI, 1.44-11.78; P = .008), and for nonrelapse mortality was 3.41 (95% CI, 1.15-10.09; P = .03) compared with low-risk patients. After a median follow-up of 5.9 years, the median survivals have not been reached for DIPSS risk groups low and intermediate-1, and were 7 and 2.5 years for intermediate-2 and high-risk patients, respectively. Thus, HCT was curative for a large proportion of patients with myelofibrosis, and post-HCT success was dependent on pre-HCT DIPSS classification.


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