Reversal of a rheologic cardiomyopathy following hematopoietic stem cell transplantation for sickle cell disease

Sachdev, Vandana; Hsieh, Matthew M.; Jeffries, Neal; Noreuil, Anna; Li, Wen; Sidenko, Stanislav; Hannoush, Hwaida; Limerick, Emily; Wilson, Delon; Tisdale, John F.; Fitzhugh, Courtney D.

doi:10.1182/bloodadvances.2019000387

Cited by 19 publications

(27 citation statements)

References 53 publications

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“…Laboratory and testing results were collected pre HPCT and annually post HPCT as per institutional standard procedures 26–28 . Patients who had at least one year of post‐HPCT data as of September 30, 2019 were included for post‐HPCT comparative analyses.…”

Section: Methodsmentioning

confidence: 99%

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

et al. 2021

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Summary Non‐myeloablative haematopoietic progenitor cell transplantation (HPCT) from matched related donors (MRD) has been increasingly utilized in sickle cell disease (SCD). A total of 122 patients received 300 cGy of total body irradiation (TBI), alemtuzumab, unmanipulated filgrastim‐mobilized peripheral blood HPC and sirolimus. The median follow‐up was four years; median age at HPCT was 29 years. Median neutrophil and platelet engraftment occurred on day 22 and 19 respectively; 41 patients required no platelet transfusions. Overall and sickle‐free survival at one and five years were 93% and 85% respectively. Age, sex, pre‐HPCT sickle complications, ferritin and infused HPC numbers were similar between graft failure and engrafted patients. Mean donor myeloid chimaerism at one and five years post HPCT were 84% and 88%, and CD3 was 48% and 53% respectively. Two patients developed grade 1 and 2 skin graft‐versus‐host disease (GVHD) with no chronic GVHD. Median days of recipients taking immunosuppression were 489; 83% of engrafted patients have discontinued immunosuppression. Haemoglobin, haemolytic parameters and hepatic iron levels improved post HPCT. Pulmonary function testing, hepatic histology and neurovascular imaging remained stable, suggesting cessation of further sickle‐related injury. Fourteen patients had children. In this largest group of adult SCD patients, this regimen was highly efficacious, well‐tolerated despite compromised organ functions pre HPCT, and without clinically significant GVHD.

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Section: Methodsmentioning

confidence: 99%

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

et al. 2021

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“…was retrospective, and one (Mah et al.) was experimental 15,31–43,45,47 . Most studies obtained written consent and approval through an institutional review board or ethics committee.…”

Section: Resultsmentioning

confidence: 99%

“…The remaining six studies did not indicate timing of the patients’ last crisis in relation to myocardial strain evaluation (Table 3). 31,33,37,43,45,46 Three studies (Covi et al., Sachdev et al., Sengupta et al.) reported myocardial strain at multiple timepoints, but only values obtained at steady state were included in this systematic review 38,43,44 .…”

Section: Resultsmentioning

confidence: 99%

“…In Covi et al. and Sachdev et al., patients were undergoing hematopoietic stem cell transplantation (HSCT); myocardial strain values reported prior to transplantation (presumed steady state) were included 38,43 …”

Section: Resultsmentioning

confidence: 99%

“…A growing number of studies have evaluated myocardial strain in individuals with SCD, but collective results have not yet been summarized. This systematic review is warranted to synthesize reported findings and remaining gaps in knowledge 15,31–47 …”

Section: Introductionmentioning

confidence: 99%

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Sickle cell disease and ventricular myocardial strain: A systematic review

Whipple

Joshi

Naik

et al. 2021

Pediatric Blood & Cancer

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Cardiac disease is the primary cause of death in sickle cell disease (SCD). Cardiac abnormalities begin in childhood and progress throughout life. Right and left ventricular (RV, LV) myocardial strain are early markers of systolic dysfunction but are not well investigated among individuals with SCD. The objectives of this review were to (1) identify all published studies that have evaluated ventricular myocardial strain, (2) summarize their values, and (3) compare findings with those obtained from controls. From search results of four electronic databases—Medline, Embase, Scopus, and Web of Science—42 potential articles were identified, of which 18 articles and 17 studies met eligibility criteria for inclusion. The evaluated studies demonstrate that RV and LV myocardial strain are generally abnormal in individuals with SCD compared with controls, despite having normal ejection/shortening fraction. Myocardial strain has been inconsistently evaluated in this population and should be considered any time an echocardiogram is performed.

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Cardiac effects 2 years after successful non‐myeloablative human leukocyte antigen‐matched related donor hematopoietic cell transplants in sickle cell disease

et al. 2023

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Reversal of a rheologic cardiomyopathy following hematopoietic stem cell transplantation for sickle cell disease

Abstract: Key Points Cardiac morphology improves significantly as early as 3 months after HSCT for SCD; these changes are maintained up to 1 year. Diastolic dysfunction is associated with mortality, and this study shows improvements in diastolic measures and other functional parameters.

Cited by 19 publications

References 53 publications

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

Non‐myeloablative human leukocyte antigen‐matched related donor transplantation in sickle cell disease: outcomes from three independent centres

Sickle cell disease and ventricular myocardial strain: A systematic review

Cardiac effects 2 years after successful non‐myeloablative human leukocyte antigen‐matched related donor hematopoietic cell transplants in sickle cell disease

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