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Outcome of Age-Adapted Approach to HLA-Identical Related Hematopoietic Stem Cell Transplantation in Severe Sickle Cell Disease: Saudi Experience
In this study, we reviewed the outcome of SCD patients who underwent transplant at our institution using standard protocols (NMA regimen in patients ≥14 years and myeloablative regimen in < 14 years) to address whether age remains a risk factor that influences HSCT outcome in SCD.Children (<14 years) with severe SCD received myeloablative conditioning using one of two regimens: first regimen was cyclophosphamide (Cy) 200mg/kg, busulfan (Bu) 16mg/kg, and thymoglobulin (ATG) 10mg/kg and recently we use thiotepa 8mg/kg, Bu 16mg/kg, and fludarabine (Flu) 160mg/m2. Bu pharmacokinetics was performed to target AUC of 900...
Source: Blood - November 21, 2018 Category: Hematology Authors: Alzahrani, M., Damlaj, M., Essa, M., Alahmari, B., Alaskar, A., Hejazi, A., Basher, E., Abujoub, R., Ghazi, S., Abuelgasim, K., Salama, H., Gmati, G., Alsultan, A. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster II Source Type: research

Efficacy and Safety of Pomalidomide in Combination with Prednisone in Patients with Myelofibrosis and Anemia -- Final Results of a Prospective Phase 2 Study
CONCLUSION:Pomalidomide with prednisone is safe therapy with good anti-anemia activity in patients with MF. It could lead to transfusion independence in one third of patients for a median duration of about 30 months. ClinicalTrials.gov Identifier: NCT00946270.Table 1.DisclosuresDaver: Alexion: Consultancy; ImmunoGen: Consultancy; Pfizer: Research Funding; Karyopharm: Research Funding; Otsuka: Consultancy; Novartis: Consultancy; ARIAD: Research Funding; Incyte: Consultancy; Pfizer: Consultancy; BMS: Research Funding; Sunesis: Research Funding; Daiichi-Sankyo: Research Funding; Sunesis: Consultancy; Kiromic: Research Funding...
Source: Blood - November 21, 2018 Category: Hematology Authors: Masarova, L., Daver, N. G., Kadia, T. M., Pemmaraju, N., Jabbour, E. J., Zhou, L., Pierce, S. A., Cortes, J. E., Kantarjian, H. M., Verstovsek, S. Tags: 634. Myeloproliferative Syndromes: Clinical: Poster I Source Type: research

Transcranial Doppler Screening Adherence Among Children with Sickle Cell Anemia Seen in the Emergency Department
Conclusion: Patients with SCA who present to the ED and are nonadherent to TCD screening guidelines are less likely to have had a recent hematology clinic visit. Therefore, the ED may be an important location for identifying patients lost to regular clinic follow-up in need of a TCD. An intervention that specifically targets this patient population will likely improve TCD screening rates and stroke prevention.Figure.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Weisman, J. K., Diamond, C., Kappa, S., Nickel, R. S. Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster II Source Type: research

Probability of TCD-Normalization in the "Drepagreffe" Trial Comparing Transplantation to Chronic Transfusion in Sickle Cell Anemia Children with Abnormal-Transcranial Doppler Is Associated with Lower Ang-2 and BDNF Plasma Levels
This study confirms the association between high levels of BDNF and high velocities, and suggests that transplantation increases the likelihood of TCD-normalization compared to transfusion, and is associated with reduced Ang-2 expression in plasma, which may reflect improved brain oxygenation.DisclosuresThuret: Addmedica: Research Funding; bluebird bio: Research Funding; Novartis: Research Funding. Pondarré: Addmedica: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria; Blue Bird Bio: Honoraria. Bernaudin: AddMedica: Honoraria; Pierre fabre: Research Funding; Cordons de Vie: Res...
Source: Blood - November 21, 2018 Category: Hematology Authors: Bernaudin, M., Khelif, Y., Chadebech, P., Bodivit, G., Jouard, A., Verlhac, S., Paillard, C., Thuret, I., Guitton, C., Runel, C., Missud, F., Arnaud, C., Kamdem, A., Pondarre, C., Pirenne, F., Bernaudin, F. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Novel or Improved Approaches To Treating Sickle Cell Disease Source Type: research

Outcomes in Children with Severe Vasculopathy from Sickle Cell Anemia after Treatment with Chronic Transfusion, Surgical Revascularization and/or Allogenic Hematopoetic Stem Cell Transplantation
Conclusions: The risk for cerebral infarction and/or vasculopathy progression after initiation of treatment with either chronic transfusion, HSCT or EDAS is still a major concern. Our data suggest HSCT and surgical revascularization with chronic transfusion provide the greatest benefit in reducing stroke risk and HSCT reduces risk for progression of a severe vasculopathy. Additional, large population studies are needed to clarify the risk.DisclosuresMajumdar: NIMHD: Research Funding. Campbell: Functional Fluitics: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on ...
Source: Blood - November 21, 2018 Category: Hematology Authors: Johnson, C. W., Majumdar, S., Campbell, A. D., Magge, S., Darbari, D. S., Webb, J., Diab, Y. A., Nickel, R. S., Speller-Brown, B., Carpenter, J., Abraham, A. Tags: 114. Hemoglobinopathies, Excluding Thalassemia-Clinical: Poster I Source Type: research

Long-term treatment follow-up of children with sickle cell disease monitored with abnormal transcranial Doppler velocities
We present the long-term follow-up of SCA children from the Créteil newborn cohort (1992-2012) detected at risk by TCD and placed on chronic transfusions. Patients with normalized velocities and no stenosis were treated with hydroxyurea, known to decrease anemia and hemolytic rate. Trimestrial Doppler was performed and transfusions restarted immediately in the case of reversion to abnormal velocities. Patients with a genoidentical donor underwent transplant. Abnormal time-averaged maximum mean velocities (TAMMV) ≥200 cm/s were detected in 92 SCA children at a mean age of 3.7 years (range, 1.3-8.3 years). No strok...
Source: Blood - April 6, 2016 Category: Hematology Authors: Bernaudin, F., Verlhac, S., Arnaud, C., Kamdem, A., Hau, I., Leveille, E., Vasile, M., Kasbi, F., Madhi, F., Fourmaux, C., Biscardi, S., Gluckman, E., Socie, G., Dalle, J.-H., Epaud, R., Pondarre, C. Tags: Pediatric Hematology, Sickle Cell Disease, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Clinical Trials and Observations Source Type: research

How I treat and manage strokes in sickle cell disease
Neurologic complications are a major cause of morbidity and mortality in sickle cell disease (SCD). In children with sickle cell anemia, routine use of transcranial Doppler screening, coupled with regular blood transfusion therapy, has decreased the prevalence of overt stroke from ~11% to 1%. Limited evidence is available to guide acute and chronic management of individuals with SCD and strokes. Current management strategies are based primarily on single arm clinical trials and observational studies, coupled with principles of neurology and hematology. Initial management of a focal neurologic deficit includes evaluation by...
Source: Blood - May 28, 2015 Category: Hematology Authors: Kassim, A. A., Galadanci, N. A., Pruthi, S., DeBaun, M. R. Tags: Sickle Cell Disease, How I Treat, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Clinical Trials and Observations Source Type: research