scholarly journals Prospective evaluation of chronic organ damage in adult sickle cell patients: A seven-year follow-up study

2017 ◽  
Vol 92 (10) ◽  
pp. E584-E590 ◽  
Author(s):  
Charlotte F. J. van Tuijn ◽  
Marein Schimmel ◽  
Eduard J. van Beers ◽  
Erfan Nur ◽  
Bart J. Biemond
Keyword(s):  
Sickle Cell ◽  
Organ Damage ◽  
Prospective Evaluation ◽  
Follow Up Study

Prospective evaluation of a chest pain pathway in the coronary care unit at green lane hospital; One year follow up study

2003 ◽  
Vol 12 (2) ◽  
pp. A34
Author(s):  
John J. Edmond ◽  
John K. French ◽  
Hanneke Henny ◽  
Ralph A.H. Stewart ◽  
Teena West ◽  
...  
Keyword(s):  
Chest Pain ◽  
Coronary Care Unit ◽  
Prospective Evaluation ◽  
Follow Up Study ◽  
Pain Pathway ◽  
Coronary Care ◽  
One Year

Therapeutic Hydroxyurea Dosing Is Associated With Decreased Organ Damage and Longer Survival In Adults With Sickle Cell Anemia

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2228-2228
Author(s):  
Courtney Fitzhugh ◽  
Darlene Allen ◽  
Wynona Coles ◽  
Michael Ring ◽  
Xiongce Zhao ◽  
...  
Keyword(s):  
Ejection Fraction ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Organ Damage ◽  
Prolonged Survival ◽  
Computer Assisted ◽  
Initial Visit ◽  
Organ Function

Abstract Introduction Hydroxyurea (HU) is the only FDA-approved medication to treat adults with sickle cell anemia (hemoglobin SS disease, SCA). Two studies showed improved survival with long term HU therapy. Conversely, subsequent studies reported most patients continue to die by the fifth decade of life despite long term treatment. Further, no studies have shown that HU prevents organ dysfunction. In this work, we sought to assess the effect of HU dosing on survival and organ function in SCA in a cohort of patients at a single referral institution. Methods Adults with SCA enrolled on the Bethesda Sickle Cell Cohort Study between January 2001 and October 2012 were included in this retrospective analysis. The NIH Biomedical Translational Research Information System was used to electronically retrieve clinical notes, administration records, and medication orders. Computer-assisted review facilitated the extraction of HU dose histories. Survival, laboratory, and cardiopulmonary parameters were compared between enrollment and most recent follow-up based on HU status and fetal hemoglobin (HbF) response. Results Of 388 subjects with SCA, 254 (65%) were treated with HU at a median dose of 19.4 mg/kg/day. Mean follow-up was 3.58±3.53 years. Of subjects prescribed HU, 166 (65%) were on a therapeutic dose. Subjects taking HU had a significantly higher HbF (9.2 versus 6.8%, p=0.0003) and mean corpuscular volume (MCV, 96.2 versus 88.3fL, p<0.001) at their initial visit compared to those not taking HU. A similar pattern was seen at the last visit for HbF (12.0 versus 6.5%, p<0.0001) and MCV (99.2 versus 87.4fL, p<0.0001), suggesting that HU was administered for a prolonged period of time. Total hemoglobin was not different between groups at either visit (8.9 versus 9.0g/dL at initial visit, p=0.41 and 8.9 versus 8.8g/dL at most recent visit, p=0.66 in HU versus no HU patients, respectively). There was no difference in overall survival based solely on HU treatment (p=0.11). However, subjects prescribed therapeutic HU doses (15-35 mg/kg/day, N=166) were more likely to be alive than subjects who never took HU (N=131, p=0.04). Survival was similar when comparing sub-therapeutic HU (N=46) to no HU (p=0.52) groups, and survival was also similar between subjects prescribed sub-therapeutic HU versus therapeutic HU (p=0.46). Multivariate analysis confirmed HU dose is independently associated with prolonged survival (p=0.006). At enrollment, creatinine was lower (1.2 versus 0.8mg/dL, p=0.0016), total bilirubin was greater (3.3 versus 2.8mg/dL, p=0.02), and tricuspid regurgitant velocity was higher (2.6 versus 2.5m/s, p=0.03) in patients on HU. There were no significant differences in ejection fraction, brain natriuretic peptide, transaminases, alkaline phosphatase, or direct bilirubin. Further, there were no differences for any markers of organ function at the most recent follow-up. To determine if the best HU responses were associated with improved organ function, subjects were divided into groups based on whether their maximum HbF was within the lowest or the highest quartile. The HU dosages were 24.1 versus 4.8mg/kg/day in the high versus low HbF groups, respectively (p<0.0001). Maximum HbF was 26.1% as compared to 1.5% in the low HbF group. Ejection fraction was higher and transaminases and creatinine were lower at both visits in subjects with the highest HbF levels (see Table). After adjusting for age, only creatinine remained lower in the high HbF group at enrollment (p=0.007). Conclusions HU therapy correlates with prolonged survival and preservation of organ function, but only when given in therapeutic dosages. These HU associations are not observed for sub-therapeutic doses less than 15 mg/kg/day. Patients should be treated with a maximum tolerated HU dose to achieve the highest possible HbF response, ideally before organ damage occurs. Additional studies are indicated to further confirm these findings. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Hypertensive organ damage predicts future cognitive performance: A 9-year follow-up study in patients with hypertension

2018 ◽  
Vol 20 (10) ◽  
pp. 1458-1463 ◽  
Author(s):  
Renske Uiterwijk ◽  
Julie Staals ◽  
Marjolein Huijts ◽  
Sander M. J. van Kuijk ◽  
Peter W. de Leeuw ◽  
...  
Keyword(s):  
Cognitive Performance ◽  
Organ Damage ◽  
Follow Up Study

Reproductive Behaviour of Couples at Risk for Sickle Cell Disease in Cuba: a Follow-Up Study

1997 ◽  
Vol 17 (8) ◽  
pp. 737-742 ◽  
Author(s):  
Aida Dorticós-Balea ◽  
Marcos Martin-Ruiz ◽  
Piedad Hechevarria-Fernández ◽  
Martha S. Robaina-Castellanos ◽  
Manuel Rodriguez-Blanco ◽  
...  
Keyword(s):  
At Risk ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Reproductive Behaviour ◽  
Cell Disease ◽  
Follow Up Study

Hydroxyurea Treatment of Young Children with Sickle Cell Anemia: Safety and Efficacy of Continued Treatment – the BABY HUG Follow-up Study

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 7-7 ◽  
Author(s):  
Zora R. Rogers ◽  
Billie Fish ◽  
Zhaoyu Luo ◽  
Rathi V. Iyer ◽  
Courtney D. Thornburg ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Hospital Admissions ◽  
Controlled Trial ◽  
Laboratory Data ◽  
Acute Chest Syndrome ◽  
Follow Up Study ◽  
Hydroxyurea Treatment ◽  
The Impact

Abstract Abstract 7 BABY HUG [Clinical Trials #NCT00006400], an NIH-NICHD sponsored randomized placebo-controlled trial showed that hydroxyurea (HU) administered to 9–18 month old children with sickle cell anemia (SCA) provides substantial clinical benefit. Benefits include a decrease in pain crises, acute chest syndrome events, need for transfusion and hospital admission; hematologic improvement include higher total and fetal hemoglobin concentration, larger red cell size, and lower WBC counts with toxicity limited to transient reduction in absolute neutrophil count (ANC) [Lancet 2011; 377:1663–72]. The parent or guardian of all 176 children who completed at least 18 months of randomized treatment were offered participation in an initial observational BABY HUG Follow-Up Study and 163 (93%) consented to participate. Clinical and laboratory data were collected every 6 months by structured abstraction of the medical record regarding use of clinically prescribed HU (dose escalation recommended), blood counts, clinical imaging, and sickle cell-related events. At the time of enrollment the family did not know their child's randomized study treatment assignment; 133 (82%) initially chose clinical prescription of open-label HU. Acceptance of HU has remained high through 36 months of follow-up; during each 6 month data collection period 68–75% of participants reported having taken HU. Only 2 patients have left the study (due to relocation) and more than 93% of expected data have been collected. Preliminary analyses as of May 2011, including 417 patient years (pt-yrs) of follow up, demonstrate that in comparison to participants not taking HU, children who continue to take HU have statistically lower rates of pain crises requiring emergency department (ED) visits, episodic transfusions, and hospital admissions for any reason, including acute chest syndrome or febrile illness (see table). The substantial decrease in acute chest syndrome episodes is similar to the effect demonstrated with HU use in the randomized BABY HUG trial in younger infants and consistent with published trials detailing the benefit of HU therapy in older children and adults. The decrease in the rate of admission for febrile events in HU-treated patients is also comparable to that in the randomized trial, but the reason for this benefit is uncertain. There was no difference in hospitalization rates for painful events including dactylitis. Two patients in the non-HU group had a stroke. There were no differences between groups in the frequency of a palpable spleen or rate of acute splenic sequestration crises. Through 36 months of follow up children taking HU had persistently higher hemoglobin and MCV, and lower WBC and ANC than those not taking HU. Results of these analyses including growth and development assessments will enhance our understanding of the impact of HU use in children with SCA starting at a very young age. The accruing data from the BABY HUG Follow-Up Study demonstrate a continuation of the substantial benefits of early HU therapy with no discernable additional toxicities. Ongoing follow up of this cohort is essential to fully define these benefits as children grow, and to observe for late toxicity.Event Rate per 100 pt-yrsHUNo HUp valueED visit for Pain Crisis28.853.60.004Episodic Transfusion18.334.00.010Hospital Admission (any cause)74.9133.20.001Acute Chest Syndrome (admission)9.522.30.0001Febrile Illness (admission)30.764.3<.001Pain Crisis (admission)18.630.40.102 Disclosures: Off Label Use: Hydroxyurea is not indicated for treatment of children with sickle cell disease. Use of this medication was for clinical indications and not mandated by this observational study.

scholarly journals A Two-Year Follow-Up Study and Prospective Evaluation of theDSM-IVAxis V

2003 ◽  
Vol 54 (7) ◽  
pp. 1028-1030 ◽  
Author(s):  
Phillipa Hay ◽  
Mary Katsikitis ◽  
Jules Begg ◽  
Jason Da Costa ◽  
Natalia Blumenfeld
Keyword(s):  
Prospective Evaluation ◽  
Follow Up Study

scholarly journals Successful Transition from Paediatric to Adult Care in Sickle Cell Disease: An Experience in a Belgian Center

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2495-2495
Author(s):  
Tracy Vandergraesen ◽  
Laurence Dedeken ◽  
Andre Efira ◽  
Phu Quoc Lê ◽  
Sophie Huybrechts ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Organ Damage ◽  
Transition System ◽  
Cell Disease ◽  
Adult Care ◽  
Walk Distance ◽  
The Us ◽  
Cardio Vascular

Abstract Introduction Despite reduction of mortality during childhood, young adults with SCD are at higher risk of morbidity and mortality after transition from paediatric to adult care, mainly in the US. To evaluate our transition system, we have studied the outcome of patients beyond the transition to adult care and looked if it was associated with an increase in vaso-occlusive crisis (VOC) and acute chest syndromes (ACS), the occurrence of progressive organ damage and changes in hydroxyurea prescription. Patients and Methods This study focus on sickle cell disease patients, born after January 1, 1980 and who have been transitioned from pediatrics (Hôpital Universitaire des Enfants Reine Fabiola, HUDERF) to adult care (CHU Brugmann), followed for minimum 2 years at HUDERF and having transitioned at least 2 years before December 2015. Demographic data, genotype, disease modify therapies, biological data, clinical data, acute clinical event, 6 minutes-walk test, cardiac echography) were collected retrospectively. Patients who underwent a hematopoietic stem cell transplantation were excluded from the analysis. Student test was used to analyse continuous variable and Wilcoxon regression for paired sample. Mc Nemar's test was used for comparison of paired nominal data and Chi-square test when data missed. Results The comparison of the data before and after transition is detailed in Table 1. Thirty eight patients (11 males) were included and made the transition at a median age of 20.1 years (range: 18.0-26.8). All patients were HbSS. The median age at diagnosis was 0.8 year (range: 0-7.95). The median duration of the follow-up before the transition was 15.95 years (range: 3.3 - 25.2) and after transition was 4.7 years (range: 2.2-5.8 years), accounting for 552.15 and 159.61 patient-years (PY) of follow-up respectively. Two patients died, 5.2 years and 0.6 year after the transition respectively. The death rate after transition was of 1.25/100 PY. Four women have delivered after a median gestation of 37.1 weeks (range: 35.9-38.7 weeks). The median new-born weight was 2.9 kg (range: 2.4-3.4 kg). The rate of disease modifying therapies remains stable after the transition. 33 patients were treated with hydroxyurea before transition compared to 35 after transition. Four patients were on chronic transfusion program before and 6 after transition. Hematological data were not statistically different at time of transition when compared to last follow-up, suggesting stable compliance to therapy but creatinine level increased significantly. VOC and ACS rate did not increased after transition as well as hospitalisation rate and days of hospitalisation. Tricuspid regurgitation velocity (TRV) increased significantly over the time but no patient developed pulmonary hypertension. The 6-minutes walk distance decreased significantly. Systolic and diastolic blood pressure increased significantly between the time of transition and the last follow-up. Regarding the educational status (8 data missing), 21 (70%) patients were able to reach graduate school and only 4 were not able to finish high school. Conclusion Our data provide useful information on the outcome during the years after transition. No increase in hospitalizations, VOC or ACS was observed. This is very different from the data provided by Blinder et al. One of the reason could be that comparing to the US situation, the Belgian Health Care System provide access and quality of healthcare even in underprivileged population. The prescription rate of HU remained unchanged and the compliance to treatment evaluated by hematological parameters remained stable. Nevertheless, changes in cardio-vascular parameters and creatinine levels suggest that despite the well-standardized follow-up for young adult patient with sickle cell disease, progressive organ damage leading to renal and cardio-vascular disease might not be prevented. However, compared to the literature, we have found less high level of TRV (9.7% vs. 24-45%), no patient with pulmonary hypertension (compared to 6-10%). In addition, our patients seems to have a better 6-minutes walk distance even in the childhood cohort (paediatrics data : 562m vs. 491m in Waltz study; adults data : 490m vs. 398m). Additional data from a larger number of patients as well as a longer period of follow-up are required to confirm the efficacy of our transition system. Disclosures No relevant conflicts of interest to declare.

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