Preliminary findings from an ongoing assessment show an allogenic leukemia-derived dendritic cell vaccine is capable of generating tumor-specific immune responses as well as potential tumor control in patients with 

American Society of Hematology (ASH) 2020 Annual Meeting

, also presented 2 incidents in which patients were converted from measurable residual disease (MRD) positive to MRD negative status, with year-plus remission duration, after the vaccination.

International investigators, led by Arjan van de Loosdrecht, MD, PhD, of Amsterdam University Medical Center, are continuing analysis of dendritic cell vaccine candidate DCP-001, for patients with AML. In phase 1 and 2 trial data, the vaccine has shown humoral and cellular immune responses capability as well as conversion of MRD positive patients.

In this current trial presented at ASH 2020, investigators are assessing the vaccine in up to 20 patients with AML who are MRD positive while in first complete remission (CR1). The patients are additionally ineligible for hematopoietic stem cell transplantation (HSCT).

DCP-001 is administered at a primary regimen of 4 times 25.10

 cells per vaccination, biweekly, followed by 2 booster vaccinations (10.10

 cells per vaccination) at weeks 14 and 18 after start of treatment.

Van de Loosdrecht and colleagues are assessing for a primary endpoint of safety and tolerability for the 2 vaccination schedules and the effect of vaccination on AML patient MRD status. They’re also seeking cellular and humoral immune response associated with DP-001, per peripheral blood assays.

As of July 15 of this year, 10 patients have been enrolled and completed a first dose cohort of 25.10

 cells per vaccination. Patient age was 41-76 years old, with gender being split half-male, half-female.

At the time of their reporting, investigators observed consistent tolerance and limited local injection site adverse events. Relapse prior to vaccination schedule completion was observed in 3 patients.

Of the 4 patients eligible for MRD evaluation, 2 became negative at the first timepoint following initial vaccinations at week 14, and remained negative until end of active follow-up, at 32 weeks.

“Two other patients remained in CR, but with MRD positivity,” investigators wrote. “For the remaining 3 patients, to date, no MRD outcome is available yet but patients remain in CR.”

Investigators stressed the small size of patients observed for immune response to DCP-001. However, they also noted one such patient showed a clinical response becoming MRD negative vaccination.

Indeed, they concluded that vaccination with DCP-001 for AML could generate a tumor-specific immune response, and eventually may provide tumor control. Long-term prevention was promising, as the 2 MRD-negative patients remained in complete remission beyond a year after vaccination.

“This study continues to enroll patients at a higher vaccine dose of 50.10

 cells per vaccination and additional data on induced immune responses and MRD status will be shown,” investigators concluded.

Conversion from MRD Positive to Negative Status in AML Patients in CR1 after Treatment with an Allogeneic Leukemia-Derived Dendritic Cell Vaccine

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New data from an ongoing trial showed patients achieved MRD negative status after a vaccine regimen—and have maintained status over a year.

Vaccine Could Provide Long-Term Acute Myeloid Leukemia Remission

Findings from a retrospective analysis reported a 7.5% prevalence of

patients. The study was conducted at 3 university health systems in the Chicago Metropolitan Area.

The investigators also evaluated and identified risk factors for thrombosis.

American Society of Hematology (ASH) 2020 Conference

, was led by Stephanie Berg, DO, Division of Hematology and Oncology, Loyola University Medical Center, and was conducted between March 2020 – May 2020.

The health systems included were the Loyola University Health System (LUHS), Rush University System for Health (RUSH), and University of Illinois-Chicago (UIC).

All patients (n = 2180) evaluated were admitted to the hospital for COVID-19. The investigators confirmed pulmonary embolism, deep-vein thrombosis, or arterial thrombosis through supportive imaging modalities.

For their analyses, they used Wilcoxon rank sum test to test the associations of continuous variables as well as Chi-square test or Fisher’s exact test to evaluate the associations of categorical variables.

Overall, they assessed a total of 2180 patients across the 3 hospital systems.

Hispanics represented 47% of patients at LUHS, 32% at RUSH, and 36% at UIC. Additionally, African Americans represented 17%, 42%, and 51%, respectively.

Of the overall population, 33% were admitted for intensive care.

A Report of Thrombotic Event Patterns Across the Hospital Systems

In terms of total thrombotic events, LUHS reported 5.4% events. Of this amount, 41 patients were reported with venous thromboembolism and pulmonary embolism, 10 with arterial thrombosis, and 5 with both venous and arterial embolism. 

RUSH reported 9.7% of thrombotic events, with 70 being classified venous thromboembolism and pulmonary embolism, 7 arterial embolism, and 4 with both venous and arterial embolism.

And finally, UIC reported a 6% rate. Of this, 14 were venous thromboembolism and pulmonary embolism, 4 were pulmonary, and none were both venous and arterial.

Berg and team noted that patients age, sex, and BMI did not differ among patients that developed a thrombotic event.

They also reported that anticoagulation prophylaxis was given to 82% of patients the LUHS and UIC at time of admission.

“Collectively, those with thrombotic events (N = 156) had higher incidence of intensive care admission, elevated white blood cell count and a d-dimer >5X upper limit normal, at presentation,” they wrote. “Furthermore, a higher proportion of patients that had a thrombotic event were diabetic at LUHS and RUSH.”

They further explained that the mortality rate in all COVID-19 patients was 13-16%. However, patients in with a thrombotic event in RUSH and UIC were noted to have an increased risk of death.

The team indicated that future studies will aim to explore the mortality differences and anticoagulation practices between the institutions as well as race/ethnicity differences in thrombosis events.

“Finally, based off these risk factors, identification of patients at most risk for thrombosis is needed to reduce the morbidity and mortality when diagnosed with COVID-19,” Berg and team concluded.

Real World Outcomes of Sars-Cov-2 Thrombosis Rates across Three University Health Systems in the Chicago Metropolitan Area

Investigators evaluated patients from 3 Chicago hospital systems and assessed risk factors associated with thrombosis. 

Study Reports on Thrombotic Rates Among COVID-19 Patients 

New data from a human cell assessment suggest a form of gene editing may eventually provide a cure of 

Results from an explorative trial, presented at the 

, evidence that knockout of Krüppel-like factor 1 (

) expression in the human cells of patients with sickle cell disease via CRISPR-based editing may provide them better regulation of their fetal hemoglobin (HbF) expression.

The Melbourne, Australia-based investigators, led by Kevin R. Gillinder, PhD, of the Australian Centre for Blood Diseases at Monash University, are continuing their assessment, but expressed confidence in the potential of targeting 

As the most common monogenic disease in humans, sickle cell disease is prevalent in millions of persons globally. The hematologic condition is driven by a homozygous T to A transcersion in the beta-globin gene that results in amino acid variant G6V and the production of HbS, which then polymerises under hypoxic conditions within red blood cells.

“This generates irreversibly sickled cells that fail to traverse the microcirculation, resulting in micro-infarcts, hypoxia and pain, or ‘sickle cell crises’,” Gillinder and colleagues wrote.

It’s during gestation that red blood cells use differing globin gene sets to produce HbF. As such, it isn’t until after a person is born, when adult hemoglobin (HbA) is first produced, that sickle cell disease can be identified.

As Gillinder and colleagues noted, this process of “hemoglobin switching”—a common pathway of sickle cell disease curative research for decades now—could be an opportunity for investigators to reactive adult cell HbF among patients with hemoglobinopathies.

—a vital erythroid transcription factor that coordinates expression of more than a thousand genes associated with the formation of adult red blood cells—is among the transcription factors which heavily influence hemoglobin switching.

“Heterozygosity for loss of function mutations in 

 leads to a significant increase in HbF that is beneficial to patients with beta thalassemia,” investigators wrote. “We propose this can be recreated by advanced gene editing techniques to provide an effective therapy for SCD.”

Investigators used CRISPR-based gene editing to knockout 

 expression in human cells, then targeting and ablating the function of the factor’s second exon.

Through RNA sequencing, the team found genes including 

, a pair of cellular adhesion molecules associated with vaso-occlusive episodes in persons, that are down-regulated in a KLF1 gene dosase-dependent manner. They believe reduced expression of both genes could provide additional benefit in treating sickle cell disease.

Additionally, investigators observed HbF levels at 40% to 60% following the engineered ablation of 

 in CD34+ cells harvested from the peripheral blood of patients with sickle cell disease undergoing transfusions.

“We will report on the results of these xenotransplantation assays,” they wrote. “Taken together these results reveal the potential utility in targeting 

Early assessment from an Australian team of investigators is targeting a pathway linked to hemoglobin expression.

Targeted Gene Editing May Provide a Sickle Cell Disease Cure

American Society of Hematology (ASH) 2020 Conference 

uncovered 2 novel gene loci that have statistical associations with fetal hemoglobin (HbF), a known ameliorator of 

A team led by Anu Marahatta, PhD, Division of Hematology, Cincinnati Children’s Hospital Medical Center, analyzed genomic DNA from children with sickle cell anemia in order to identify novel genes and variants involved in HbF expression.

“Previous genomic studies have identified three major gene loci (BCL11A, HBS1L-MYB, and HBG2) that account for ~40% of HbF variation in SCA, but additional genetic modifiers remain to be discovered,” the team wrote.

All patients (n = 1009) assessed had pre-treatment steady-state HbF levels and were enrolled in various prospective research trials from the United States, the Caribbean, and sub-Saharan Africa.

In these trials, whole blood DNA were genotyped and many samples underwent whole exome sequencing (WES) to identify over 2.2 million single nucleotide variants across the genome.

Marahatta and colleagues proceeded in 3 different phases for their genome wide association study (GWAS).

First, they performed 2 independent GWAS discovery steps using distinct African populations. They designated these steps as Discovery I (n= 211) and Discovery II (n = 223).

From both datasets, they selected for 2 independent replications steps only the single nucleotide variants that were significant (

 < .05). They then designated these single nucleotide variants as either African-American (n = 157) or African (N = 269).

Finally, any single nucleotide variants that were significant in the first phase (dual discovery) and at least 1 one of the replication cohorts were then verified using an additional Caribbean cohort (N=149) with TaqMan techniques for genotyping specific variants.

Old and New Genomic Loci Are Associated with HbF Expression

From this process, the team was able to search for genomic loci with consistent HbF association across various cohorts.

“From the combined SNP and WES dataset, 8 

variants passed genome wide significance (

) in the discovery analysis, and 1,048 additional variants were identified with nominal HbF association (

However, following phase 2, they found that 173 of the novel variants had sustained association in at least of the replication cohorts (

After selecting 20 variants with the strongest and most consistent associations with HbF from the discovery and replication analyses, the team was able to identify and verify the HbF associations with BCL11A (rs1427407) and HBS1L-MYB (rs4895441).

Among the 18 potentially novel variants, the rs77737207 variant near the RUNX1T1 locus was strongly associated with lower HbF levels. A similar association, which approached statistical significance (

 < .08) in the final verification cohort, was also noted for the coding variant rs2279587 in the ITGA1 gene.

“The RUNX1T1 gene is a broad transcriptional corepressor known to impact myeloid differentiation in hematopoiesis, while ITGA1 encodes the integrin alpha subunit of a cell-surface receptor involved in cell-cell adhesion and inflammation,” the team wrote.

They indicated that both genes may be involved in this regulation of HbF expression in the pediatric sickle cell anemia population. Marahatta and colleagues suggested that future studies should investigate them further using cellular and animal models.

Novel Genetic Loci That Influence Fetal Hemoglobin Expression in Children with Sickle Cell Anemia

High levels of fetal hemoglobin have previously been linked to the mitigation of morbidity and mortality of sickle cell anemia. 


Novel Genetic Loci May Be Linked with HbF Expression in Sickle Cell Children

CPX-351 used in combination with venetoclax was tolerable in patients with 

American Society of Hematology (ASH) 2020 virtual conference

Furthermore, the investigators reported that virtually all responders were cleared to move on to stem cell transplant.

A team led by Tapan M. Kadia, MD, Department of Leukemia, MD Anderson Cancer Center, Houston, Texas, evaluated the safety and efficacy of the combination therapy in patients with Relapsed/Refractory AML.

Previous research has shown CPX-351, a liposomal formulation of cytarabine, to lead to higher response rates, better tolerability, and survival in patients with secondary AML when compared with 7+3. Further, addition of venetoclax to hypomethylating agents has also been shown to lead to greater improvement in response rate and survival than homethylating agents alone.

Kadia and team enrolled 18 patients with a median age of 51 years. Overall, 94% of patients had Relapsed/Refractory AML, and the median number of prior therapy was 2.

More than half (67%) were assigned to the lead-in safety cohort, which received 300 mg as the starting effective dose of venetoclax. Dosage was administered between days 2 – 21, and interruption of venetoclax was permitted after day 14 if bone marrow hypocellular and had no evidence of leukemia.

After safety was established, the investigators developed two more cohorts. Cohort A (28%) included patients with Relapsed/Refractory AML, and Cohort B included only 1 patient with newly diagnosed AML.

For both cohorts, dosage of CPX-351 was constant on days 1, 3 and 5 of induction and days 1 and 3 of consolidation.

Reported Responses of Combination CPX-351 and Venetoclax 

Of the 16 evaluated patients with Relapsed/Refactroy AML, 1 experienced a complete remission, 5 reached complete remission with incomplete hematologic recovery, and 1 reached a morphologic leukemia-free state. Thus, the overall response rate was 44%.

The investigators also noted that the overall response rate for patients without any prior exposure to venetoclax was 60% (6/10)—versus 17% (1/6) among those with prior exposure.

Additionally 86% of responding patients (6/7) went on to receive stem cell transplant.

The median length of overall survival was 6.4 months, and the 6-month survival rate was 53%.

Among those who responded to the combination treatment, the median 6-month survival and relapse-free survival rate were both 86%.

The 4- and 8-week mortality rates were 11% and 22% respectively.

“The starting dose level -1 was above the maximum tolerated dose of the combination; dose-limiting toxicities were prolonged neutropenia and thrombocytopenia,” they wrote.

The most frequent serious adverse events rated grades 3 and 4 were infection, nausea, pneumonia, and myelosuppression.

“CPX-351 plus 7 days of venetoclax is tolerable, with acceptable toxicities in patients with R/R AML. Within this high risk AML cohort, CPX + venetoclax demonstrated encouraging activity, particularly in those who had not received prior venetoclax,” Kadia and colleagues concluded.

They indicated that their study will continue to enroll relapsed and frontline cohorts.

Phase II Study of CPX-351 Plus Venetoclax in Patients with Acute Myeloid Leukemia (AML)

Those without prior exposure to venetoclax experienced the most benefit. 


CPX-351 Plus Venetoclax Tolerable in Patients with Acute Myeloid Leukemia

 patients need to be particularly careful during the coronavirus disease 2019 (COVID-19) pandemic.

These patients have become particularly susceptible to suffering from more severe outcomes should they contract the virus.

While there is hope for that more protections in the form of vaccines could be on the way, there is still a need to do a better job of protecting this patient population.

 Stefan Bornstein, MD, Professor of Endocrinology, Metabolism, and Diabetes at the Technical University at Dresden, explained what researchers have learned about the interaction between the COVID-19 virus and diabetic patients and what the best practices are for treating these patients.

Bornstein presented new data during the 18th World Congress of Insulin Resistance Diabetes & Cardiovascular (WCIRDC) Online CME Conference, sponsored by the Metabolic Institute of America (TMIOA).

Bornstein’s presentation focuses on the severity of the viral infections and hyperglycemia and uncontrolled diabetes. He explained it is still unknown why there is such an association between this patient population and COVID-19 outcomes.

He also said there needs to be better ways to treat the severely ill patients once they are admitted into the hospital.

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Diabetic patients are more susceptible to severe COVID-19 outcomes.

Stefan Bornstein, MD: Diabetic Patients During COVID-19

A pair of new therapies have emerged that could be crucial in treating patients who do not respond to or tolerate statin therapy as a 

—bempedoic acid and PCSK9 siRNA (inclisiran).

During the 18th World Congress of Insulin Resistance Diabetes & Cardiovascular (WCIRDC) Online CME Conference presented by the Metabolic Institute of America (TMIOA), Kausik Ray, MD, a professor of Public Health at the School of Public Health of Imperial College London presented new data showing the benefits of these new treatments.

Currently, bempedoic acid is currently approved in Europe and the US, making it the first cholesterol lowering oral agent approved in approximately 10 years.

However, inclisiran represents an entirely new class of drugs that targets a process called gene silencing. Ray expects this treatment to be available in both North America and Europe in 2021.

, how exactly these drugs work, what the current data says about their efficacy and safety, and how these drugs might be used in the future.

Ray said the importance of having new options available to reduce cholesterol is that many patients could need significant reduction in cholesterol, meaning combining therapies could be the right approach for many.

Dr. Kausik Ray presents new data on inclisiran and bempedoic acid as cholesterol lowering medications.

Kausik Ray, MD: Promising New Cholesterol Lowering Agents

A combination of infliximab with a plant-based diet as first-line therapy for patients with severe 

 has promise in increasing remission rates and lowering colectomy rates, according to a new study. This new therapy may potentially be superior to traditional corticosteroid therapy.

A prospective single-group, non-randomized, open-label, uncontrolled trial led by Mitsuro Chiba, MD, PhD, Gastroenterology Division, Ankita City Hospital, Japan, enrolled patients with severe ulcerative colitis, which they defined according to the Truelove and Witts Criteria. 

The team evaluated the outcomes of this new therapeutic approach as first-line therapy. They noted that first-line use of infliximab has been scarcely investigated.

Thus, they administered to patients infliximab (5.0 mg/kg-7.5 kg) as induction therapy at weeks 0, 2, and 6 weeks. In addition to this, all patients received a semivegetarian diet over the course of their treatment.

“To our knowledge, no previous study has incorporated a plant-based diet in the induction phase of treatment of severe ulcerative colitis,” Chiba and colleagues wrote. “We designed infliximab and a plant-based diet as first-line (IPF) therapy not for corticosteroid-refractory patients but for new patients with severe ulcerative colitis without prior intensive intravenous corticosteroid use.”

The primary endpoint sought by the investigators was clinical remission or colectomy in the induction phase and 1 year post-discharge. For the induction phase, they defined remission as the disappearance of bloody stool at week 6 following commencement of the first infliximab infusion.

Secondary endpoints included changes in inflammatory markers in the induction phase as well as the plant-based diet score—an indicator of adherence—at baseline and follow-up.

Of the total assessed (n = 17), 11 were male, and the mean age was 43 (with a range of 18-78).

In the induction phase, the remission rate was 76%, and the colectomy rate was 6%.

Further, the investigators reported that, at week 6, C-reactive protein values decreased from 9.42 mg/dL to 0.33 md/dL. Additionally, erythrocyte sedimentation rate decreased as well from 59 mm/h to 17 mm/h (

A total of 14 proceeded to the follow-up study—the other 3 discontinued due to moving elsewhere or undergoing a colectomy.

In this cohort, the cumulative relapse rate was 25%—further, there were no reported additional colectomy cases.

Mean PBD improved at year 1 (27.7) for 10 patients and year 4 (23.8) for 8 patients when compared with baseline scores of 8.3 and 9.9, respectively (p < 0.0001 and p = 0.0391)

In terms of safety, no patient in the overall study period experienced infusion reactions to infliximab. However, there were 2 cases in which metronidazole was withdrawn—1 due to hand paresthesia, the other due to gastric distress. Additionally, 5-aminosalicyic acid was withdrawn in another case due to gastroenteritis.

No adverse events—such as gaseous distress, abdominal discomfort, or diarrhea—were experienced as a result of the plant-based diet.

They acknowledged the lack of a control group as well as the small sample size were notable limitations. Nonetheless, they expressed optimism in the potential implications of their findings.

“Infliximab and a plant-based diet as first-line therapy provided better short-term and medium-term outcomes for patients with severe ulcerative colitis than the outcomes reported in the literature,” they wrote.

They indicated a few advantages to this therapy over an intensive corticosteroid regimen—including the ability for most patients to eat dinner on same day as treatment, its safety, and the overall health benefits provided by plant-based diets.

High Remission Rate with Infliximab and Plant-Based Diet as First-Line (IPF) Therapy for Severe Ulcerative Colitis: Single-Group Trial

This combination therapy may have a few advantages over standard corticosteroid regimens. 


Infliximab Plus Plant-Based Diet Efficacious, Safe for Severe Ulcerative Colitis

The US Food and Drug Administration (FDA) has approved berotralstat (ORLADEYO) for the prevention of hereditary angioedema (HAE) attacks in adults and pediatrics aged 12 years and older.

The once-daily oral therapy from BioCryst Pharmaceuticals becomes the first of its kind indicated for HAE attack prophylaxis.

Pivotal phase 3 data from the APeX-2 supporting berotralstat for the new indication showed a 150 mg dose of the therapy was associated with a significantly reduced risk of HAE attack at 24 weeks, which was sustained through the full 48-week treatment period.

Among patients treated with berotralstat for the full 48 weeks, mean monthly HAE attack decreased from 2.9 to 1.0. In a long-term open-label assessment of the same patient population, investigators observed a decrease to just 0.8 attacks monthly.

In both studies, the therapy was associated with known safety and tolerability, with the most frequently reported treatment-related adverse events being gastrointestinal reactions.

The historic indication for berotralstat provides confidence in clinicians and patients seeking to make progress in reduced HAE attack risks—without the burden of infusion or injection therapy, APeX-2 trial investigator Marc Riedl, MD, professor of medicine and clinical director at the US Hereditary Angiodema Association Center at the University of California, San Diego, said.

“Patients and physicians acknowledge that HAE treatments can add a burden to patients’ lives,” Riedl said in a 

 “With this new treatment option, physicians and patients can continue to have collaborative discussions to choose the treatment that meets each patient’s needs, life circumstances and preferences.”

Jon Stonehouse, president and chief executive officer of BioCryst, echoed sentiment on the significance of the approval.

“The FDA approval of ORLADEYO fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks,” he said. “We will stay focused on enabling access and providing personalized support to HAE patients and physicians.”

The indication is based on 48-week results showing significant benefit in reduced HAE attacks among pediatric and adult patients.

FDA Approves Berotralstat as First Oral Hereditary Angioedema Prophylaxis

Findings from a new study demonstrated that hematopoietic stem cell transplantation (HSCT) for patients with 

 was not associated with a significant increase in 

A team led by Emily Limerick, MD, The Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins Medicine, evaluated the prevalence of chronic kidney disease prior to and following operation as well as the incidence of acute kidney injury within days of HSCT.

“Though HSCT can reverse the SCD phenotype, acute and chronic kidney dysfunction may be an unintended consequence,” the team wrote. “The incidence of acute kidney injury (AKI) and the effect of different HSCT regimens on progression or amelioration of CKD in adults with SCD have not previously been reported.”

Thus, the cohort study analyzed prospectively collected data from 106 patients with sickle cell disease who had undergone HSCT at the National Institutes of Health (NIH) Clinical Center.

More than half (n = 71) of the patients had an HLA-matched sibling. A smaller proportion had haploidentical HSCT (N=26) where nonmyeloablative conditioning was given, and fewer underwent gene therapy (N=9) where a myeloablative regimen was employed.

All patients’ renal function were assessed at baseline and annually for 3 years.

Acute kidney injury was defined and evaluated according to Kidney Disease: Improving Global Outcomes (KDIGO) criteria.

At baseline, the prevalence of chronic kidney disease 6% (eGFR <60 ml/min/1.73m

) — this prevalence did not change significantly post-HSCT.

However, the investigators noted that when defined less conservatively (urine albumin to creatinine ratio (UACR) ≥30 mg/g; or eGFR <60 ml/min/1.73m

), chronic disease prevalence at baseline was 48%, followed by 62% at year 1 (

“The incidence of CKD any time after HSCT was 1% and 11% according to each of these CKD definitions, respectively,” the investigators wrote.

Furthermore, they reported that baseline median eGFR was 139.7 ml/min/1.73m

 and declined by 7.6, 11.6, and 20.9 ml/min/1.73m2 each year post-HSCT (

Hyperfiltration (eGFR ≥150 ml/min/1.73m

) was present in 34% of patients at baseline; however, that steadily declined to 12% after 3 years following operation. (

Overall, patients with normal eGFR (60-149 ml/min/1.73m

) increased from 60% at baseline to 80% at 3 years post-HSCT.

There was no noted association of worsening eGFR with gender, engraftment status, transplant regimen, or conditioning agent (each variable, 

And finally, 58% of patients experienced acute kidney injury in the 1st 100 days following transplantation. Over that subset, 77% were mild, 13% were moderate, and 10% were severe. The investigators considered these findings to be consistent with previous data.

Thus, Limerick and colleagues underscored the preservation of renal function following hematopoietic stem cell transplantation.

Renal Function Is Preserved after Hematopoietic Stem Cell Transplantation for Sickle Cell Disease

Patients with normal eGFR increased from 60% at baseline to 80% at 3 years following procedure.

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