Autologous bone marrow transplant in a patient with sickle cell disease and diffuse large B-cell lymphoma

Onitilo, Adedayo A.; Lazarchick, John; Brunson, Chris Y.; Frei‐Lahr, Debra; Stuart, Robert K.

doi:10.1016/j.transproceed.2003.10.085

Cited by 9 publications

(6 citation statements)

References 22 publications

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“… 9 – 10 Between 2003 and 2008, eight other patients requiring autologous HSCT for a malignant hematopoietic disease were mobilized with Filgrastim after additional precautions had been taken: reduction of the HbS level to below 30% via red blood cell exchange, and carefully monitoring of peripheral leukocytosis and the blood ion profile. 11 – 13 Although all eight patients experienced bone pain, hypertension or migraine, no severe adverse events were reported - providing evidence that SCD patients can be mobilized without any major complications. The median [interquartile range] circulating CD34 + cell count was 24.4/μL [21.2–48.6].…”

Section: Introductionmentioning

confidence: 76%

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

et al. 2018

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Sickle cell disease is characterized by chronic anemia and vaso-occlusive crises, which eventually lead to multi-organ damage and premature death. Hematopoietic stem cell transplantation is the only curative treatment but it is limited by toxicity and poor availability of HLA-compatible donors. A gene therapy approach based on the autologous transplantation of lentiviral-corrected hematopoietic stem and progenitor cells was shown to be efficacious in one patient. However, alterations of the bone marrow environment and properties of the red blood cells hamper the harvesting and immunoselection of patients’ stem cells from bone marrow. The use of Filgrastim to mobilize large numbers of hematopoietic stem and progenitor cells into the circulation has been associated with severe adverse events in sickle cell patients. Thus, broader application of the gene therapy approach requires the development of alternative mobilization methods. We set up a phase I/II clinical trial whose primary objective was to assess the safety of a single injection of Plerixafor in sickle cell patients undergoing red blood cell exchange to decrease the hemoglobin S level to below 30%. The secondary objective was to measure the efficiency of mobilization and isolation of hematopoietic stem and progenitor cells. No adverse events were observed. Large numbers of CD34+ cells were mobilized extremely quickly. Importantly, the mobilized cells contained high numbers of hematopoietic stem cells, expressed high levels of stemness genes, and engrafted very efficiently in immunodeficient mice. Thus, Plerixafor can be safely used to mobilize hematopoietic stem cells in sickle cell patients; this finding opens up new avenues for treatment approaches based on gene addition and genome editing. Clinicaltrials.gov identifier: NCT02212535.

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Section: Introductionmentioning

confidence: 76%

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

et al. 2018

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“…[2][3][4] Many of these malignancies may benefit from autologous hematopoietic stem cell transplantation. However, filgrastim administered to mobilize hematopoietic stem cells may precipitate severe and sometimes fatal sickle crisis in patients with sickle-cell anemia or complex sickle-cell hemoglobinopathies.…”

mentioning

confidence: 99%

“…[5][6] A previous single report describes successful autologous marrow transplantation in a patient with sickle-cell disease and diffuse large-cell lymphoma. 2 Filgrastim was used to support initial chemotherapy and was also administered post transplantation without clinical evidence of sickle-cell crisis. We now report a successful filgrastim mobilized hematopoietic stem cell transplantation for diffuse largecell lymphoma in a patient with sickle-cell hemoglobin C disease.…”

mentioning

confidence: 99%

Successful mobilization and transplantation of filgrastim mobilized hematopoietic stem cells in sickle cell-hemoglobin C disease

Kamble

Tin-U

Carrum

2006

Bone Marrow Transplant

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“…With survival into adulthood, patients with SCA are susceptible to comorbidities including heart disease, cognitive decline, pulmonary hypertension, venous thromboembolism, complications of pregnancy and transfusional iron overload, and others . In addition, recent studies indicate an upward of twofold excess of cancer, in particular hematological malignancies, in patients with sickle cell disease . In the treatment of many hematological malignancies, consolidation of the response to chemotherapy with high‐intensity chemotherapy and autologous hematopoietic progenitor cell (HPC) rescue has become the standard of care .…”

Section: Introductionmentioning

confidence: 99%

“…One report describes a 40‐year‐old woman with SCA and diffuse large B‐cell lymphoma who underwent high‐dose chemotherapy and autologous bone marrow rescue . She received 20 days of filgrastim after the HPC infusion.…”

Section: Introductionmentioning

confidence: 99%

Automated red blood cell exchange in preparation for filgrastim mobilization of autologous peripheral blood hematopoietic progenitor cells in a patient with sickle cell anemia

Zhao

Bailey

Linden

et al. 2017

J of Clinical Apheresis

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Increasing survival of patients with sickle cell anemia (SCA) well into adulthood results in a rising likelihood of developing hematological malignancy. High-dose chemotherapy with autologous hematopoietic progenitor cell (HPC) rescue is standard of care for several hematological malignancies, but the risk of severe or life-threatening vaso-occlusive phenomena during filgrastim mobilization of HPC for collection poses a potential barrier to this approach. We report the use of automated red cell exchange in preparation for filgrastim mobilization in a patient with homozygous SCA. Red cell exchange was repeated just prior to high-dose chemotherapy to mitigate the need for red cell transfusion during bone marrow reconstitution. The patient experienced no vaso-occlusive phenomena throughout the entire episode of care and did not become iron overloaded. This approach should be considered for all patients with homozygous or compound heterozygous sickle cell disease who are candidates for auto-HPC rescue therapy.

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Autologous bone marrow transplant in a patient with sickle cell disease and diffuse large B-cell lymphoma

Cited by 9 publications

References 22 publications

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

Successful mobilization and transplantation of filgrastim mobilized hematopoietic stem cells in sickle cell-hemoglobin C disease

Automated red blood cell exchange in preparation for filgrastim mobilization of autologous peripheral blood hematopoietic progenitor cells in a patient with sickle cell anemia

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