Normalized Transcranial Doppler Velocities, Stroke Prevention and Improved Pulmonary Function after Stem Cell Transplantation in Children with Sickle Cell Anemia

Mynarek, Martin; Riehm, C. Bettoni da Cunha; Brinkmann, Folke; Weißenborn, Karin; Tell-Lüersen, M.; Heuft, Hans‐Gert; Maecker‐Kolhoff, Britta; Sykora, Karl‐Walter

doi:10.1055/s-0033-1333754

Cited by 14 publications

(16 citation statements)

References 23 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…4 Similar results involving other centers, [5][6][7] as well as long-term follow-up studies of transplanted patients, have demonstrated that HSCT definitively cures SCD, preventing further, and in some cases even reversing, SCD-mediated organ damage. [8][9][10] More recent studies of HLA-identical sibling HSCT for SCD have reported even better results, with overall survival approaching 100% and SCD cure rates of ;90%.…”

Section: Introductionmentioning

confidence: 99%

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Nickel

Hendrickson

Haight

2014

Blood

View full text Add to dashboard Cite

Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). HSCT using an HLA-identical sibling donor is currently an acceptable treatment option for children with severe SCD, with expected HSCT survival >95% and event-free survival >85%. HSCT for children with less severe SCD (children who have not yet suffered overt disease complications or only had mild problems) is controversial. It is important to consider the ethical issues of a proposed study comparing HLA-identical sibling HSCT to best supportive care for children with less severe SCD. In evaluating the principles of nonmaleficence, respect for individual autonomy, and justice, we conclude that a study of HLA-identical sibling HSCT for all children with SCD, particularly hemoglobin SS and Sβ0-thalassemia disease, is ethically sound. Future work should explore the implementation of a large trial to help determine whether HSCT is a beneficial treatment of children with less severe SCD.

show abstract

Section: Introductionmentioning

confidence: 99%

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Nickel

Hendrickson

Haight

2014

Blood

View full text Add to dashboard Cite

show abstract

“…Due to the lack of donor availability, matched unrelated donor (MUD) marrow transplants have not been performed in sufficient number of patients with SCD. There is one report from Germany that included two children who received matched unrelated marrow transplant successfully [67]. The Sickle Cell Unrelated Transplant (SCURT) trial (NCT00745420), which has been open since 2008, has transplanted close to 20 patients from MUD (European Bone Marrow Transplant meeting 2012).…”

Section: Matched Unrelated Donor Transplantation For Patients With Hementioning

confidence: 99%

Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

Fitzhugh¹,

Abraham²,

Hsieh³

2017

Advances in Experimental Medicine and Biology

View full text Add to dashboard Cite

Considerable progress with respect to donor source has been achieved in allogeneic stem cell transplant for patients with hemoglobin disorders, with matched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many studies have solidified hematopoietic progenitors from matched sibling marrow, cord blood, or mobilized peripheral blood as the best source-with the lowest graft rejection and graft versus host disease (GvHD), and highest disease-free survival rates. For patients without HLA-matched sibling donors, but who are otherwise eligible for transplant, fully allelic matched unrelated donor (8/8 HLA-A, B, C, DRB1) appears to be the next best option, though an ongoing study in patients with sickle cell disease will provide data that are currently lacking. There are high GvHD rates and low engraftment rates in some of the unrelated cord transplant studies. Haploidentical donors have emerged in the last decade to have less GvHD; however, improvements are needed to increase the engraftment rate. Thus the decision to use unrelated cord blood units or haploidentical donors may depend on the institutional expertise; there is no clear preferred choice over the other. Active research is ongoing in expanding cord blood progenitor cells to overcome the limitation of cell dose, including the options of small molecule inhibitor compounds added to ex vivo culture or co-culture with supportive cell lines. There are inconsistent data from using 7/8 or lower matched unrelated donors. Before routine use of these less matched donor sources, work is needed to improve patient selection, conditioning regimen, GvHD prophylaxis, and/or other strategies.

show abstract

“…This has led to the investigation of unrelated and mismatched donors as a donor source (Table I). Based on case report data and promising experience in other diseases, using a fully HLA-matched unrelated donor is believed by many to be the next best HSCT option if a matched sibling is not available [29, 30]. A multicenter Clinical Trials Network (CTN) study evaluating matched unrelated donor bone marrow transplants for pediatric patients with SCD has recently completed accrual.…”

Section: Current State Of Hematopoietic Stem Cell Transplantation Formentioning

confidence: 99%

Cellular therapy for sickle cell disease

2016

View full text Add to dashboard Cite

Sickle cell disease (SCD) is a monogenic red cell disorder affecting over 300,000 annual births worldwide and leading to significant organ toxicity and premature mortality. While chronic therapies such as hydroxyurea have improved outcomes, more durable therapeutic and curative options are still being investigated. Newer understanding of the disease has implicated invariant NKT (iNKT) cells as a critical immune profile that potentiates SCD. Hence, targeting this cell population may offer a new approach to disease management. Hematopoietic stem cell transplant (HSCT) is a curative option for patients with SCD, but the under-representation of minorities on the unrelated donor registry means that this is not a feasible option for >75% of patients. Work in this area has therefore focused on increasing the donor pool and decreasing transplant-related toxicities to make this a treatment option for the majority of patients with SCD. This review focuses on the currently available cell and gene therapies for patients with SCD, and acknowledges that newer gene editing approaches to improve gene therapy efficiency and safety are the next wave of potentially curative approaches.

show abstract

Normalized Transcranial Doppler Velocities, Stroke Prevention and Improved Pulmonary Function after Stem Cell Transplantation in Children with Sickle Cell Anemia

Abstract: HSCT is able to prevent stroke in patients with SCA. Its perspectives and limitations should be discussed early during the treatment of a patient with complicated SCA.

Cited by 14 publications

References 23 publications

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease

Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

Cellular therapy for sickle cell disease

Contact Info

Product

Resources

About