scholarly journals Incorporating novel approaches in the management of MDS beyond conventional hypomethylating agents

Hematology ◽  
2017 ◽  
Vol 2017 (1) ◽  
pp. 460-469 ◽  
Author(s):  
Olatoyosi Odenike
Keyword(s):  
Critical Appraisal ◽  
Stem Cell Transplant ◽  
Clinical Investigation ◽  
Dna Methyltransferases ◽  
Cell Transplant ◽  
Dna Hypomethylation ◽  
New Agents ◽  
Hypomethylating Agent ◽  
The Us ◽  
History Of

Abstract In the last decade, the treatment of higher-risk myelodysplastic syndromes (MDS) has revolved around the azanucleosides, azacitidine and decitabine, which at lower doses are postulated to work predominantly via their effects on inhibition of DNA methyltransferases and consequent DNA hypomethylation. For patients who relapse after, or do not respond to, hypomethylating agent therapy, the outcome is dismal, and new agents and approaches that have the potential to alter the natural history of these diseases are desperately needed. Allogeneic stem cell transplant is the only known potentially curative approach in MDS, but its applicability has been limited by the advanced age of patients and attendant comorbidities. There is now an increasing array of new agents under clinical investigation in MDS that aim to exploit our expanding understanding of molecular pathways that are important in the pathogenesis of MDS. This review focuses on a critical appraisal of novel agents being evaluated in higher-risk MDS that go beyond the conventional hypomethylating agent therapies approved by the US Food and Drug Administration.

Real world treatment patterns in chronic myeloid leukemia patients newly initiated on tyrosine kinase inhibitors in an U.S. integrated healthcare system

2017 ◽  
Vol 24 (4) ◽  
pp. 253-263
Author(s):  
Nazia Rashid ◽  
Han A Koh ◽  
Kathy J Lin ◽  
Brian Stwalley ◽  
Eugene Felber
Keyword(s):  
Chronic Myelogenous Leukemia ◽  
Index Date ◽  
Stem Cell Transplant ◽  
Study Time ◽  
Myelogenous Leukemia ◽  
Prior History ◽  
Cell Transplant ◽  
Time Period ◽  
History Of

Purpose To evaluate treatment patterns in patients diagnosed with incident chronic myelogenous leukemia (CML) newly initiating therapy with imatinib, dasatinib, or nilotinib. Patients were followed to determine switching and discontinuation rates. Factors associated with switching or discontinuation from index TKI therapy, reasons for discontinuation based on electronic chart notes, and frequency of laboratory monitoring were assessed during the follow-up period. Methods A retrospective cohort study was conducted in chronic myelogenous leukemia patients aged ≥ 18 years who were identified from the Kaiser Permanente Southern California (KPSC) Cancer Registry database during the study time period of 1 January 2007 to 12 December 2013. The index date was defined as the date of the first TKI prescription (imatinib, dasatinib, or nilotinib) identified during the study time period with no prior history of TKI use within 12 months. Patients had to have continuous membership with drug benefit eligibility and no prior history of stem cell transplant (SCT) or other cancers during the 12 months prior to the index date. Baseline characteristics were identified during 12 months prior to the index date and outcomes were identified during the follow-up period after the index date. All patients were followed from index TKI therapy until end of study time period (12 December 2014), death, stem cell transplant, or disenrollment from the health plan unless one of the following occurred first: a patient switched their index therapy, or a patient discontinued their index therapy. Forward stepwise selection multivariable logistic regression models were used to evaluate factors associated with patients who continued therapy compared to those who switched or discontinued therapy with the index TKI. Chart notes were reviewed 30 days prior and 30 days post index TKI discontinuation to evaluate reasons for discontinuation. Molecular and cytogenetic testing frequency was also assessed during the follow-up period among the different patient groups. Results Two hundred sixteen patients were identified with incident chronic myelogenous leukemia and use of TKI therapy: 189 (87.5%) received imatinib, 19 (8.8%) received dasatinib, and 8 (3.7%) received nilotinib. The mean age on index date was 53 years and 63% were male; 103 patients (48%) continued on their index therapy, while 62 patients (28%) switched, and 51 patients (24%) discontinued.

Single-Agent Lenalidomide for Patients with Relapsed/Refractory Aggressive Non-Hodgkin's Lymphoma Who Had Received Prior Stem Cell Transplant.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2699-2699
Author(s):  
Julie M. Vose ◽  
Thomas Habermann ◽  
Myron S Czuczman ◽  
Pier Luigi Zinzani ◽  
Craig B. Reeder ◽  
...  
Keyword(s):  
Stem Cell ◽  
Research Funding ◽  
Stem Cell Transplant ◽  
Cell Lymphoma ◽  
Prior History ◽  
Cell Transplant ◽  
Phase 2 ◽  
Two Phase ◽  
Phase Ii Studies ◽  
History Of

Abstract Abstract 2699 Poster Board II-675 Introduction: High-dose chemotherapy with autologous stem cell transplant (SCT) is a standard treatment option for younger patients with aggressive non-Hodgkin's lymphomas (a-NHL) who fail to respond, or relapse after initial therapy. For patients who relapse after SCT there are few effective treatment options and prognosis remains poor (Vose et al Blood. 80(8):2142–8, 1992). A second SCT was shown to achieve only marginal responses and at an increased risk of toxic death (Lenain et al Hematol J. 5(5):403–9, 2004). Therefore, new approaches are needed for treatment of patients in this poor prognostic group. Lenalidomide (Revlimid®) is an immunomodulatory agent that has shown clinical activity in treatment of several B-cell malignancies. Two phase 2 studies of lenalidomide monotherapy in patients with relapsed or refractory (R/R) a-NHL were conducted in the US (NHL-002; Wiernik et al JCO 26:4952–7, 2008) and internationally (CC-5013 NHL-003). In an extended follow-up of the NHL-002 study, the overall response rate (ORR) was 35%, which included 12% of patients with a complete response (CR), and a median duration of response (DR) lasting 10.4 months (Wiernik et al 2008 EHA. Abst: 764). Comparable efficacy was observed in the larger confirmatory, phase 2, NHL-003 study of lenalidomide in a similar patient population. The goal of this analysis was to learn the ORR and DR to lenalidomide in patients with a prior SCT. Methods: Patient data from both phase II studies were pooled for this report. Eligibility for both studies was similar – patients with R/R a-NHL, which included diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), transformed lymphoma (TL), and follicular lymphoma grade III (FL-III), with measurable disease (≥ 2 cm), and ≥ 1 prior treatment regimen. Patients received oral lenalidomide 25 mg once daily on days 1–21 of a 28-day cycle. Protocol defined treatment continued for up to 52 wks in NHL-002, or until disease progression in NHL-003. Primary endpoint was ORR; secondary endpoints included DR, progression-free survival (PFS), and safety. Results: 87 patients with a prior SCT (14 patients from NHL-002; 73 patients from NHL-003) were included in this analysis. Median age of patients was 61 yrs (range, 23–76), with a median of 4 prior therapies (range, 2–12). The ORR to lenalidomide was 39% (34/87), with 13% (11/87) CR/CRu and 26% (23/87) partial response (PR). Median PFS for all 87 patients was 3.8 months (95% CI, 2.6, 5.6) and the DR for 34 responders was 9.7 months (95% CI, 4.2, 16.3). Responses occurred in 15 of 52 patients (29% ORR; 10% CR/CRu) with DLBCL, 12 of 19 patients with MCL (63% ORR; 26% CR/CRu), and in 6 of 10 patients with TL (60% ORR; 10% CR/CRu). The table summarizes responses for patients who did not have a transplant, for those who had a SCT anytime prior to lenalidomide; as the last therapy prior to lenalidomide; and not as last therapy prior to lenalidomide. Most common grade 3 or 4 adverse events were neutropenia (44%), thrombocytopenia (33%), and anemia (9%). Eighteen (20.6%) patients discontinued treatment due to adverse events. Patients with a prior history of SCT appeared to have a significantly higher rate of thrombocytopenia, all grades (51% v 30%; P = 0.001), and grades 3 or 4 (33% v 16%; P = 0.002). Patients with a prior SCT were also more likely to experience a dose reduction/interruption due to thrombocytopenia compared with those without a prior SCT (25% v 14%; P = 0.027). Conclusions: Based on a pooled dataset from two phase II studies, oral lenalidomide monotherapy appears to be a well tolerated and active therapy resulting in durable responses in patients with R/R a-NHL who had a prior SCT. Furthermore, the potential of achieving a response to lenalidomide appears to be independent of prior history of SCT. Disclosures: Vose: Celgene: Research Funding. Off Label Use: Lenalidomide for the treatment of relapsed/refractory aggressive non-Hodgkin's lymphoma. . Czuczman:Celgene: Research Funding. Zinzani:Celgene: Research Funding. Tuscano:Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees. Pietronigro:Celgene: Employment, Equity Ownership. Ervin-Haynes:Celgene: Employment. Witzig:Celgene: Research Funding.

Disparities in the Upfront Use of Hematopoietic Stem Cell Transplant Among Patients with Acute Myeloid Leukemia

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3543-3543
Author(s):  
Tahir Mehmood ◽  
Adam C Bartley ◽  
Shahrukh K. Hashmi ◽  
Ronald S. Go
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Myelogenous Leukemia ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
The Us ◽  
Risk Patients ◽  
Good Risk

Abstract Background: The incidence for acute myelogenous leukemia (AML) is on the rise in the United States (US). Roughly 20,000 new AML patients are expected to occur in 2016 in the US (data from Surveillance Epidemiology and End Results Program). It is mainly a disease of older individuals with median age of 67 years. Unfortunately, most patients are not cured. Recent data suggest that early referral for hematopoietic stem cell transplant (HSCT) leads to more favorable outcomes, particularly those with non-good risk cytogenetics compared to alternate therapies. The objective of our study was to assess the use of upfront HSCT among AML patients with a focus on patients with non-good risk cytogenetics and determine disparity in its access based on sociodemographic and regional considerations. Methods: We analyzed data obtained from National Cancer Data Base (NCDB) Participant User Files for patients diagnosed with AML between 2004 and 2013 using the International Classification of Diseases for Oncology version 3 (ICD-O-3) codes 9840-9861, 9865-9874, 9891-9931. The data is collected prospectively by joint program of Commission on Cancer (CoC) and American Cancer Society. It covers nationwide oncology outcome from more than 1,500 CoC-accredited hospitals. Over 70% of all newly diagnosed cancer cases in the US are captured by NCDB. Since unique ICD-O-3 codes exist for the good-risk but not for intermediate- or poor-risk AML, we categorized the AML subtypes as good risk or non-good risk for analytic purposes. We considered patients with t(15;17), inv(16) and t(8;21) with corresponding ICD-O codes of 9871, 9896, and 9866, as good-risk patients. All others were considered non-good risk. Proportion of HSCT usage was estimated in various subgroups with 95% confidence intervals calculated using robust estimates of standard error. Results: We identified 80,087 patients who were diagnosed with AML between 2004 and 2013. Majority of these patients were Whites (85.9%), males (53.8%) and between the ages of 40 and 79 years (71.5%). The overall use of upfront HSCT increased consistently over time. This occurred predominantly among the non-good risk AML from a rate of 6.5% in 2004 to 12.5% in 2013 (Figure). Non-good risk patients who were Black, had Medicare insurance, with lower annual household income, and treated at facilities located in the East, South and Central parts of US ( AL, KY, MS, TN) had less access to HSCT (Table). Conclusion: The use of upfront HSCT in AML has almost doubled in the past decade, predominantly among those with non-good risk cytogenetics. Nevertheless, we found substantial disparity among sociodemographic and geographic subgroups. Future studies should try to understand and address how to bridge this gap. Disclosures No relevant conflicts of interest to declare.

scholarly journals A Population-Based Study of Chronic Myelomonocytic Leukemia in the United States from 2004-2015

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 30-31
Author(s):  
Gordon Ruan ◽  
Caleb J. Smith ◽  
Courtney Day ◽  
William Harmsen ◽  
Abhishek A. Mangaonkar ◽  
...  
Keyword(s):  
Stem Cell ◽  
Myeloid Leukemia ◽  
Stem Cell Transplant ◽  
Population Based ◽  
Cell Transplant ◽  
Monocytic Leukemia ◽  
Academic Center ◽  
The Us ◽  
Myelomonocytic Leukemia

Background: Chronic myelomonocytic leukemia (CMML) is a rare chronic myeloid malignancy with a poor prognosis. There are few epidemiologic studies on CMML. We studied population-based outcomes in CMML using the Surveillance, Epidemiology, and End Results (SEER) and the National Cancer Database (NCDB). Methods: The SEER 18 registries and NCDB Participant User File were used to identify patients with ICD-O-3 diagnosis code 9945/3 from 2004-2015. Incidence was identified using SEER and age-adjusted to the U.S. 2000 standard population. Causes of death were obtained and CMML-related death was defined as death from any myeloid disorder (CMML, acute monocytic leukemia, acute myeloid leukemia [AML], chronic myeloid leukemia, other myeloid/monocytic leukemia, and aleukemic, subleukemic, and NOS) to avoid misattribution. Relative survival (RS) was defined as the ratio of the proportion of observed survivors in a cohort of CMML patients to the proportion of expected survivors in a comparable set of individuals that did not have CMML, adjusting for the general survival of the US population for race, sex, age, and time when the diagnosis was established. Time to AML was calculated using the left-truncated life tables session and a 3 month latency period was used to prevent misattribution. Per the NCDB's system on classifying treatment, hydroxyurea, decitabine, and azacitidine are considered chemotherapy. The Kaplan-Meier method was used to calculate overall survival (OS), and Cox regression model was used to identify predictors of survival. Variables significant in univariate analysis (age, Charlson Deyo score [CDS], insurance type, and treatment facility type) were included in a multivariate analysis. Statistical analyses were performed using SAS version 9.0. Results: In the SEER database (n=4437), the median age at diagnosis was 76 years (interquartile range [IQR] 68-83) and 2,783 patients (63%) were male. Incidence rates (1 case/1,000,000 individuals) were as follows: overall 4.4, male 6.6, female 2.9, Non-Hispanic White 4.9, and Non-Hispanic Black 3.3. The incidence did not significantly change from 2004 to 2015. With a median follow up of 5.8 years (95% CI: 5.5-6.3), the median OS was 1.3 years (95% CI: 1.3-1.4). 3635 patients (82%) died. Among those who died, 2016 patients (55%) deaths were CMML-related. When comparing CMML patients to the US general population, 3,156 patients were matched to the expected survival tables. In the general U.S. population, the expected survival for 12, 24, 36, 48 and 60 months was 95.6%, 91.3%, 87.1%, 83.1%, and 79.2%, respectively. In contrast, the RS for patients with CMML at the same time points was 63.6%, 46.2%, 35.2%, 28.8%, and 23.1% (Figure 1). 229 patients (5.2%) progressed to AML. The median time to AML was 1.2 years (IQR 0.6-2.3). In NCDB (n=6403), the median age at diagnosis was 74 years (IQR 66-82). With the median follow up of 6 years (95% CI 3.5-9), the median OS was 1.3 years (95% CI 0.4-3.3). The distribution of CDS score 0, 1, and >1 was 4518 (71%), 1193 (18%), and 692 (11%) respectively. 4687 (77%) had government insurance, while 1399 (23%) had private insurance. 3750 (60%) were treated at a non-academic center. The year of diagnosis was associated with improved OS (HR 0.99, 95% CI: 0.98-0.99). Patients who were diagnosed in 2012-2015 had improved OS compared to patients diagnosed in 2004-2007 (HR 0.89, 95% CI 0.83-0.95) with OS at 1- and 5- years being 60% and 16% (2012-2015) vs. 56% and 17% (2008-2011) vs. 53% and 15% (2004-2007) respectively. 3029 (48%) patients received chemotherapy, while 270 (4%) patients received stem cell transplant as the first-line therapy. The median time to chemotherapy from diagnosis was 19 days (IQR 6-43). OS at 1-, 5-, and 10-years was 57%, 18%, and 4% (no chemotherapy), 56%, 14%, and 7% (received chemotherapy), 79%, 44%, and 38% (received stem cell transplant), and 56%, 15%, 5% (no stem cell transplant). OS curves are shown in Figure 2. Factors independently predicting inferior OS were age ≥ 65 years at the time of diagnosis (p<0.001), CDS ≥ 1 (p<0.001), government insurance (p<0.001), and treatment at a non-academic center (p<0.001) (Table). Conclusion: Despite an improvement in survival over the years, CMML continues to be a cause of significant morbidity and mortality. Older age, CDS ≥ 1, government insurance, and treatment at non-academic facilities were predictive of inferior survival. Disclosures Shah: Dren Bio: Consultancy.

scholarly journals The Incidence and Associated Risk Factors in Central Venous Catheter Associated Infectious Complications within the Autologous Stem Cell Transplant Population of Eastern Health

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 31-31
Author(s):  
Tom Dunne ◽  
David M. Jones
Keyword(s):  
Risk Factors ◽  
Stem Cell ◽  
Incidence Rate ◽  
Infectious Complication ◽  
Stem Cell Transplant ◽  
Infectious Complications ◽  
Eastern Health ◽  
Cell Transplant ◽  
Factors Associated ◽  
History Of

Background: Central venous catheters (CVCs) carry a risk of infectious complication with associated morbidity and mortality. Hematologic malignancies are a known independent risk factor for these complications. Patients undergoing hematopoietic stem cell transplant (HSCT) are at increased infectious risk with an incidence of 24.7-31.3%. Proposed contributors to this risk are the underlying malignancy, extended neutropenia, and increased requirement for blood products. Existing cohort studies of HSCT patients have been unable to identifying consistent, modifiable risk factors to target with infection prevention and control initiatives. This single-centre retrospective cohort study examines an autologous stem cell transplant population to identifying risk factors associated with CVCs. Objectives:To determine the incidence and incidence rate per 1000 catheter-days within the autologous HSCT program at Eastern HealthTo identify protective and risk factors associated with CVCs in HSCT patientsDescribe the causative agents in CVC infectious complication identified by blood and catheter-tip culture results Methods: Charts of all adult patients with hematologic malignancy who underwent HSCT with CVC placement at Eastern Health between January 1, 2014 and March 1, 2020 were examined to determine which patients experienced any of a catheter-related bloodstream, tunneled-line or exit site infectious complication as defined by the Public Health Agency of Canada's surveillance definitions. Risk factors assessed included patient factors (age, malignancy, history of bacteremia, fungemia or radiation therapy), immunosuppressive factors (lines of chemotherapy, total 90 day corticosteroid burden, erythropoietin use, days to polymorphonuclear cells >500/µL), and CVC factors (line type, insertion site, antibiotic prophylaxis, heparin impregnation, training level of radiologist, days indwelling, thrombotic complication. Additional data captured included 90-day mortality, whether the CVC was terminated and the causative organism identified by blood or catheter-tip culture. Preliminary Results: The incidence of total infectious complications was 56.2% with an incidence of catheter-related bloodstream infection (CR-BSI) of 22.9%. The incidence rate for total infectious complications was 6.51 per 1000-catheter days, with a CR-BSI infectious rate of 2.65. Both incidence and incidence rate were below results found in other centres. In univariate analysis found single-line of chemotherapy (HR 0.114, p=0.001), use of Permacath (HR 0.03, p=0.002), right internal jugular placement (HR 0.048, p=0.006) to be protective for infectious complication. Multivariate analysis identified a history of bacteremia (OR 763.1, p=0.039) and total days CVC indwelling (OR 0.94, p=0.011) to be associated with CVC infectious complication. Conclusion: Modifiable risk factors associated with CVC infectious complication are choice of device and placement site. Risk factors such as multiple lines of previous chemotherapy or history of bloodstream infection signal need to increased observation for infection. Disclosures No relevant conflicts of interest to declare.

scholarly journals Early Detection of Vulvovaginal Graft-Versus-Host Disease: An Integrative Review

2021 ◽  
Vol 12 (7) ◽  
Author(s):  
Payton L. Ten Hagen, DNP, APRN, FNP-C ◽  
Christi Bowe, MSN, APRN, ANP-C ◽  
Joyce E. Dains, DrPH, JD, APRN, FNP-BC, FNAP, FAANP
Keyword(s):  
Stem Cell ◽  
Early Identification ◽  
Stem Cell Transplant ◽  
Small Sample ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Host Disease ◽  
Female Patients ◽  
Presenting Symptoms ◽  
History Of

Introduction: Vulvovaginal graft-vs.-host disease (VVGvHD) is a condition caused by a T-cell mounted immune response after allogeneic hematopoietic stem cell transplant (alloHSCT), which can lead to sclerotic changes of the external genital organs. A common complication of alloHSCT, VVGvHD is underreported and underdiagnosed in female patients. Without detection and treatment, VVGvHD can progress to complete obliteration of the vaginal canal requiring surgical intervention in severe cases. Design: This review summarizes findings to assist providers in detecting and treating VVGvHD. It utilized PubMed, Scopus, and CINAHL databases. Inclusion criteria consisted of female patients, a history of stem cell transplantation, and a history of VVGvHD. Studies not published in English and dated more than 15 years were excluded. After the evaluation of 333 articles, 10 were included based on relevance and applicability. Limitations of this review included small sample sizes, retrospective nature of articles, and lack of randomized control trials. Findings: Early identification of VVGvHD requires identifying the rate of occurrence and risk factor profile, recognizing the presenting symptoms, improving VVGvHD assessment techniques, ascertaining when to biopsy, and establishing clinically targeted surveillance programs. Conclusion: For female patients who have undergone alloHSCT, targeted surveillance for early identification of VVGvHD results in earlier treatment initiation. Subsequently, this can improve sexual health, partner relationships, and quality of life in patients after stem cell transplant.

Long Term Prospective Follow-Up after Treatment Intensification in Pediatric Sickle Cell Patients: Comparative Effects of Transfusion Program (TP), Hydroxyurea (HU) or Stem Cell Transplant (SCT) on Annual Check-Ups.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3196-3196
Author(s):  
Francoise Bernaudin ◽  
Emmanuelle Lesprit ◽  
Lena Coïc ◽  
Cécile Arnaud ◽  
Emmanuelle Fleurence ◽  
...  
Keyword(s):  
Pediatric Patients ◽  
Stem Cell Transplant ◽  
Young Patients ◽  
Biological Data ◽  
Cell Transplant ◽  
Treatment Intensification ◽  
Old Patients ◽  
History Of

Abstract Treatment intensifications in SCD with HU, TP or SCT are applied in order to reduce SCD related complications but their comparative effects have still to be described. We report our experience concerning the annual check-up performed in SCD pediatric patients. Patients and Methods: Among our cohort of 397 SS/Sb0 pediatric SCD patients, 157 of them were intensified with HU (n= 86), TP (n=104) or SCT (n=36) and some of them received successively HU, TP and SCT. HU was proposed to patients > 3 years of age and having experienced more than 3 VOC/ACS/year or < 7g/dl severe anemia. TP defined as > 4 months program was applied in patients with cerebral vasculopathy defined by an history of stroke or abnormal TCD (> 200 cm/sec). TP was also proposed in patients with HU-failure and in patients with frequent VOC, less than 3 years old. SCT was proposed in patients with an indication of treatment intensification and an available HLA identical sibling donor. Annual check-up were performed in our day-care unit. We analysed 1261 check-ups performed and recorded since 1992 in 341 SS/Sb0 patients (sex: 164 F, 177 M). Median age was 8.8 ± 5.1 years. Mean number of annual check-ups per patient was 3.7 ± 2.8 (range 1 to 13): 816 were performed in non intensified patients, 196 in HU, 123 in TP and 126 in transplanted patients. Categories of age were distinguished: < 2 y of age (n=110), 2–5y (n=244), 5–10y (n=415), 10–15y (n=317) and 15–20y (n=175). Results: Respective follow-up were 4.4 y ± 3.3 in HU, 2.6 y ± 2.6 inTP and 5.8 y ± 4.7 in SCT patients. Comparison with non intensified patients showed that weight was significantly higher in SCT patients > 15 y of age (p=0.001), spleen size was significantly higher in (2–5y) young patients treated with HU (p=0.005) or TP (0.001) and in 5–10 y old patients on HU (p=0.046) but no difference was observed after the age of 10 y. O2 saturation was significantly improved after SCT (p<0.001) (98.8 ± 1.0 vs 97.1 ± 2.6) and was unchanged on HU and TP. Cardiac pulsations were significantly (p<0.001) decreased after all type of intensification. Biological data are shown (table1and 2). Conclusion : Treatment intensifications (TP, HU, SC) reduced the decrease of weight observed with aging in SCD patients and significantly reduced anemia using different mechanisms. SCT was the most effective to correct anemia, supress hemolysis and decrease leucocytosis. Intensif. n Follow-up HbF% Eryht Hb MCV Retic mean (SD) No 816 11.4 (9.2) 3.1 (0.9) 8.1 (1.2) 81.4 (8.9) 268.9 (105.2) HU 196 4.4 y. (3.3) 13.9 (7.0) 2.7 (0.6) 8.5 (1.2) 97.7 (13.7) 188 (83.8) TP 126 2.6 y.(2.7) 3.3 (3.1) 3.1 (0.6) 9.1 (1.4) 86.8 (4.8) 258.2 (126.0) SCT 123 5.8 y.(4.7) 4.6 (6.4) 4.3 (0.9) 11.4 (1.6) 81.5 (8.9) 89.4 (63.4) Intensif. n Tot Bili Conj Bili LDH Ferritin Leucocytes Platelets No 816 49.8 (34.4) 5.7 (3.4) 1016 (312) 192 (322) 13.2 (9.9) 385 (124) HU 196 47.5 (34.4) 5.0 (2.2) 943 (264) 399 (582) 9.7 (3.8) 352 (133) TP 126 58.8 (39.6) 5.6 (2.2) 973 (377) 2238 (6310) 13.1 (4.7) 365 (128) SCT 123 15.6 (13.9) 2.8 (4.2) 493 (200) 1099 (1386) 6.8 (3.3) 295 (109)

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