scholarly journals Torquetenovirus Dynamics and Immune Marker Properties in Patients Following Allogeneic Hematopoietic Stem Cell Transplantation: A Prospective Longitudinal Study

2018 ◽  
Vol 24 (1) ◽  
pp. 194-199 ◽  
Author(s):  
Philipp Wohlfarth ◽  
Michael Leiner ◽  
Christian Schoergenhofer ◽  
Georg Hopfinger ◽  
Irene Goerzer ◽  
...  
Keyword(s):  
Stem Cell ◽  
Longitudinal Study ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Prospective Longitudinal Study ◽  
Hematopoietic Stem ◽  
Immune Marker ◽  
Prospective Longitudinal

scholarly journals Long-Term Neurodevelopmental Outcomes of Hematopoietic Stem Cell Transplantation for Late-Infantile Krabbe Disease

Blood ◽  
2020 ◽  
Author(s):  
Isabel C Yoon ◽  
Nicholas A Bascou ◽  
Michele D Poe ◽  
Paul Szabolcs ◽  
Maria L Escolar
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Symptom Onset ◽  
Disease Onset ◽  
Krabbe Disease ◽  
Hematopoietic Stem ◽  
Prospective Longitudinal

Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in galactocerebrosidase. The only effective treatment is hematopoietic stem cell transplantation (HSCT). Approximately 85% of Krabbe cases are the infantile subtypes, among which ~20% are late-infantile. Prior studies demonstrated that HSCT is effective for early-infantile patients (0 - 6 months of age) transplanted while asymptomatic, compared to those transplanted while symptomatic. However, no studies evaluated the efficacy of HSCT for late-infantile patients (6 - 36 months). In this prospective, longitudinal study, patients were evaluated at a single site following a standardized protocol. Survival analysis was performed using the Kaplan-Meier method. Differences between groups were estimated using Mixed Regression models to account for within person repeated measures. Nineteen late-infantile patients underwent HSCT (1997 - 2020). Compared to untreated patients, transplanted patients had a longer survival probability and improved cognitive and language function. Gross and fine motor development were most affected with variable results. Asymptomatic patients benefitted the most from transplantation with normal to near-normal development in all domains and some gross motor delays. Among symptomatic patients, those with disease onset > 12 months of age had better cognitive outcomes than untreated patients. Those with disease onset £ 12 months were comparable to untreated patients. We found that HSCT prolongs the lifespan and improves the functional abilities of late-infantile Krabbe patients, particularly for those transplanted before symptom onset. In addition, our findings support prior literature that reclassifies late-infantile Krabbe disease to be symptom onset 12 to 36 months of age.

Patient Characteristics Associated With Receiving Hematopoietic Stem Cell Transplantation In An Older, Transplant-Appropriate MDS Cohort: The MDS-TAO Study

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2929-2929
Author(s):  
Gregory A. Abel ◽  
Haesook T. Kim ◽  
David P. Steensma ◽  
Angel M Cronin ◽  
Kristofer D. Earles ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Performance Status ◽  
Ecog Performance Status ◽  
Hematopoietic Stem ◽  
Fatigue Score ◽  
Prospective Longitudinal

Abstract Background The factors that influence utilization of reduced-intensity conditioning hematopoietic stem cell transplantation (RIC HSCT) for “fit” elderly patients with advanced myelodysplastic syndromes (MDS) remain unclear. Methods The “MDS Transplant-Associated Outcomes Study,” or “MDS-TAO,” is a prospective longitudinal observational study which began at the Dana-Farber/Harvard Cancer Center in May of 2011. It is designed to examine survival, quality of life (QoL), and other outcomes for RIC HSCT versus non-HSCT approaches for HSCT-appropriate MDS patients ages 60 to 75. Inclusion criteria include: histologically-confirmed diagnosis of MDS or CMML, and at least one of the following: (1) therapy-related disease, or (2) intermediate-2/high risk IPSS (Greenberg, 1997), or (3) intermediate/poor-prognosis risk cytogenetics (Haase, 2007), or (4) severe and sustained anemia or thrombocytopenia, or (5) platelet or red cell transfusion dependence. Exclusions include: (1) comorbidities that in the judgment of the enrolling clinician preclude HSCT eligibility (2) prior donor search, and (3) patient unwillingness to consider HSCT. For this analysis, time to HSCT was estimated using Kaplan-Meier methods, and log rank tests were used to assess time to HSCT by age, gender, ECOG performance status, IPSS, IPSS cytogenetic risk group, and baseline EORTC QLQ-C30 global QoL and fatigue scores. Results As of April 30, 2013, 87 patients had been enrolled. The median age was 69 years, 66% were male, and 88% had an ECOG performance status of 0 or 1. As of July 15, 2013, 22 had received HSCT within a median of 4.0 months (range 2-10 months) from study enrollment, and 17 had died without receiving HSCT. The 9-month probability of having had a transplant was 31% (95% CI [21% to 44%]). The median global QoL score was 66.7 and the median fatigue score was 33.3 (published medians are 66.7 and 33.3 for other cancers; higher global QoL scores indicate superior QoL whereas higher fatigue scores indicate worse fatigue). MDS-TAO patients with poorer cytogenetics (p<.001) and worse IPSS at enrollment (p<.001) were more likely to undergo HSCT. Age showed a complex relationship (p=.03), with those aged 65-70 most likely to undergo HSCT (34%), followed by those aged 60-65 (33%), and those aged >70 (11%). Female gender (p=.10), performance status (p=.14), global QoL (p=.56; see figure), and fatigue score (p=.58; see figure) all showed no significant association with the likelihood of undergoing HSCT. Conclusion Our data suggest that while older transplant-appropriate MDS patients suffer from similar QoL impairment as compared to other cancer patients, cytogenetics and IPSS likely have more influence than patient-reported QoL in influencing which patients ultimately receive HSCT. Disclosures: No relevant conflicts of interest to declare.

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