A systematic review comparing allogeneic hematopoietic stem cell transplant to gene therapy in sickle cell disease

Rotin, Lianne E; Viswabandya, Auro; Kumar, Rajat; Patriquin, Christopher J.; Kuo, Kevin H.M.

doi:10.1080/16078454.2022.2163357

Cited by 7 publications

(5 citation statements)

References 99 publications

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“…Considering that gene therapies are currently being introduced in the treatment of hemoglobinopathies for patients lacking a suitable donor, and that these therapies also use a fully myeloablative preparative regimen, it is highly relevant in the field of cellular therapies to identify the most tolerable option able to achieve durable engraftment and disease‐free outcomes 11,13 . As the long‐term efficacy and safety of gene therapies for hemoglobinopathies have as yet not been established, the importance of refining HCT procedures in their treatment is mandatory, because HCT is likely to remain crucial to cure patients with hemoglobinopathies with an available MRD 41 . A treatment with a non‐myeloablative conditioning (non‐MAC) regimen was developed by the National Institutes of Health (NIH) and has been evaluated by various groups to assess its outcome 20,21,42 .…”

Section: Discussionmentioning

confidence: 99%

“…11,13 As the long-term efficacy and safety of gene therapies for hemoglobinopathies have as yet not been established, the importance of refining HCT procedures in their treatment is mandatory, because HCT is likely to remain crucial to cure patients with hemoglobinopathies with an available MRD. 41 A treatment with a non-myeloablative conditioning (non-MAC) regimen was developed by the National Institutes of Health (NIH) and has been evaluated by various groups to assess its outcome. 20,21,42 The NIH protocol uses alemtuzumab (1 mg/kg) in combination with 300 cGy TBI, along with sirolimus as GvHD prophylaxis.…”

Section: Discussionmentioning

confidence: 99%

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Good engraftment after reduced intensity targeted busulfan‐based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies

Klink,

Felber,

Zeilhofer

et al. 2024

Pediatric Blood & Cancer

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BackgroundHematopoietic cell transplantation (HCT) is an established curative therapy for transfusion‐dependent thalassemia (TDT) and sickle cell disease (SCD). The latest American Society of Hematology guidelines recommend myeloablative preparative regimen in patients under 18 years of age.ProcedureThe objective was to demonstrate safety and efficacy of a reduced intensity conditioning (RIC) regimen including high‐dose fludarabine, anti‐thymocyte globulin, and targeted busulfan as a single alkylator to sub‐myeloablative exposures.ResultsBetween 2012 and 2021, 11 patients with SCD and five patients with TDT and matched related donor (MRD) HCT were included. The median age at transplantation was 8.3 years (range: 3.7–18.8 years). The median administered busulfan AUC was 67.4 mg/L×h (range: 60.7–80 mg/L×h). Overall survival was 93.8% and event‐free survival 87.5% with one engrafted SCD patient with pre‐existing moyamoya disease succumbing after drainage of a subdural hematoma. One SCD patient developed a secondary graft failure and was treated with a second HCT. Myeloid chimerism was full in all other patients with a median follow‐up time of 4.1 years (range: 2.0–11.1 years), whereas T‐cell donor chimerism was frequently mixed.ConclusionThis RIC conditioning followed by MRD HCT is sufficiently myeloablative to cure pediatric patients with hemoglobinopathies without the need for additional total body irradiation or thiotepa.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Good engraftment after reduced intensity targeted busulfan‐based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies

Klink,

Felber,

Zeilhofer

et al. 2024

Pediatric Blood & Cancer

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“…Following transplantation, donor hematopoietic stem cells replace those of the host (though some recipients continue to have both donor and recipient blood cells) [22]. Prior studies of myeloablative and nonmyeloablative stem cell transplantation have shown long-term event-free survival rates of near or above 90% following transplantation [23,24]. Many prior studies have utilized matched sibling donors, which significantly limits the number of patients who are eligible for this treatment.…”

Section: New and Emerging Therapiesmentioning

confidence: 99%

Systemic medications for sickle cell disease and potential applications for sickle cell retinopathy

Garg,

Scott

2024

Current Opinion in Ophthalmology

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Purpose of review To review the literature evaluating systemic medications for treatment of sickle cell disease (SCD) and their applications for sickle cell retinopathy. Recent Findings Prior studies have demonstrated the efficacy of traditional systemic therapies in reducing the risk of development of sickle cell retinopathy. Since 2017, several new and promising disease-modifying therapies for sickle cell disease have been approved for clinical use, including the first genetic therapies such as exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel). These treatments have shown promising results for systemic management but are not widely utilized due to limited access and high cost. The efficacy of these therapies for the prevention of sickle cell retinopathy remains unknown and opens the door to new avenues for research. Furthermore, the role of systemic therapy for the management of hemoglobin SC (HbSC) disease, which has milder systemic effects but higher likelihood of causing retinopathy, remains poorly understood. Summary Hydroxyurea has been a mainstay of systemic management of SCD with prior work suggesting its ability to reduce the likelihood of developing retinopathy. There are several new and potentially curative systemic therapies for SCD, though their role in retinopathy prevention and management has not been studied extensively. Future studies are necessary to understand the implications of these emerging therapies for sickle cell retinopathy.

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“…Traditional cancer treatment methods involve chemotherapy, surgery, and radiation therapy, which can somewhat alleviate the condition but often come with side effects. New noninvasive cancer treatment methods use specific physical and chemical means to target and destroy cancer or tumor cells without affecting other healthy cells, including immunotherapy, [108] gene therapy, [109] molecular targeted therapy, [110] hyperthermia, [111] radiofrequency ablation, [112] and others. Compared to traditional treatment methods such as chemotherapy, surgery, and radiation therapy, they are more effective with fewer side effects, can improve treatment efficiency, reduce side effectsHyperthermia has been a newly emerging method for treating of tumors and cancer in recent years, which has significant value in improving the treatment effect of traditional treatment methods.…”

Section: Photothermal Anti-tumor and Cancer Treatmentmentioning

confidence: 99%

Photothermal Conversion of Hydrogel‐Based Biomaterial

Wei

et al. 2023

The Chemical Record

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Traditional energy from fossil fuels like petroleum and coal is limited and contributes to global environmental pollution and climate change. Developing sustainable and eco‐friendly energy is crucial for addressing significant challenges such as climate change, energy dilemma and achieving the long‐term development of human society. Biomass hydrogels, which are easily synthesized and modified, have diverse sources and can be designed for different applications. They are being extensively researched for their applications in artificial intelligence, flexible sensing, biomedicine, and food packaging. The article summarizes recent advances in the preparation and applications of biomass‐based photothermal conversion hydrogels, discussing the light source, photothermal agents, matrix, and preparation methods in detail. It also explores the use of these hydrogels in seawater desalination, photothermal therapy, antibacterial agents, and light‐activated materials, offering new ideas for developing sustainable, efficient, and advanced photothermal conversion biomass hydrogel materials. The article concludes with suggestions for future research, highlighting the challenges and prospects in this field and paving the way for developing of long‐lasting, efficient energy materials.

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A systematic review comparing allogeneic hematopoietic stem cell transplant to gene therapy in sickle cell disease

Cited by 7 publications

References 99 publications

Good engraftment after reduced intensity targeted busulfan‐based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies

Good engraftment after reduced intensity targeted busulfan‐based conditioning and matched related donor hematopoietic cell transplantation in hemoglobinopathies

Systemic medications for sickle cell disease and potential applications for sickle cell retinopathy

Photothermal Conversion of Hydrogel‐Based Biomaterial

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