Experience with stem cell collection for children with Transfusion-dependent thalassemia undergoing gene therapy
This study reviews stem cell collection in 13 children with transfusion-dependent thalassemia undergoing gene therapy, highlighting that most achieved adequate CD34+ cell yields through multiple collections without adverse reactions, and emphasizing the need to improve mobilization regimens and perform saturated transfusions prior to collection.
To explore the characteristics and strategies of stem cell collection in children with Transfusion-dependent thalassemia (TDT) undergoing gene therapy. To summarize the challenges encountered and the countermeasures taken during stem cell collection in 13 TDT children undergoing gene therapy at the Department of Pediatrics, 923rd Hospital of the Joint Logistics Support Force of PLA between August 2020 and January 2024. Among the 13 TDT children, one required 1 collection, nine required 2 collections, one required 3 collections, and one required 4 collections. Adequate cells were collected in 12 cases, while 1 case had failed to obtain sufficient CD34+ cells despite multiple rounds of collection. The stem cell collection process was smooth in all 13 cases, with no adverse reactions. The stem cell mobilization regimen for TDT children needs to be enhanced, and saturated blood transfusion should be performed prior to stem cell collection.
- Abstract
- 10.1182/blood.v124.21.3856.3856
- Dec 6, 2014
- Blood
Efficacy and Risk Factors Analysis of Upfront Autologous Stem Cell Mobilization Using Plerixafor and Granulocyte-Colony Stimulating Factor (GCSF) in Patients with Multiple Myeloma
- Research Article
22
- 10.1111/j.1537-2995.2007.01448.x
- Aug 21, 2007
- Transfusion
The successful mobilization and collection of hematopoietic stem cells are dependent on a number of clinical factors such as previous chemotherapy and disease stage. The aim of this retrospective study was to determine whether the effectiveness of mobilization and collection is an independent prognostic factor for autologous stem cell transplantation outcome. A total of 358 patients who received transplants from January 2003 to December 2004 (201 male and 157 female patients, ages from 2.7 to 77.3 years with median of 53 years of age) underwent autologous hematopoietic stem cell collection after mobilization with granulocyte-colony-stimulating factor (G-CSF) or G-CSF plus chemotherapy priming. This retrospective study included patients with diagnoses of acute myelogenous leukemia, non-Hodgkin's lymphoma, Hodgkin's disease, multiple myeloma, and solid tumors. All patients underwent stem cell collection until a target or a minimum CD34+ cell dose was reached. Correlations were performed between stem cell mobilization and/or collection efficacy and transplantation outcomes. In general, both larger reinfused CD34+ cell dose and shorter number of days for the stem cell count to reach the minimum of 2 x 10(6) per kg CD34+ cells do not foster quicker engraftment. Reinfused CD34+ cell dose of less than 12 x 10(6) and number of days stem cell collection to reach this minimum CD34+ cell dose did not independently affect the overall survival (OS) or disease-free survival (DFS). The effectiveness of hematopoietic stem cell mobilization and collection as defined as number of days to reach a CD34+ cell dose of 2 x 10(6) per kg should not be used independently to forecast posttransplantation prognosis, engraftment, DFS, and OS.
- Research Article
11
- 10.1016/j.jad.2020.12.089
- Dec 28, 2020
- Journal of Affective Disorders
Construction of an exposure-pathway-phenotype in children with depression due to transfusion-dependent thalassemia: Results of (un)supervised machine learning.
- Research Article
- 10.1182/blood-2025-4185
- Nov 3, 2025
- Blood
Efficacy of motixafortide plus G-CSF versus plerixafor plus G-CSF for stem cell mobilization and collection in multiple myeloma: A single-center comparative analysis and interim Results from a prospective study in poor mobilizers
- Abstract
- 10.1182/blood-2024-204388
- Nov 5, 2024
- Blood
Stem Cell Mobilization Yields with Daratumumab (Dara) and Lenalidomide (Len)-Containing Quadruplet Induction Therapy in Patients with Newly Diagnosed Multiple Myeloma (NDMM): A Real-World Experience at Two Institutes
- Research Article
2
- 10.1016/j.clml.2025.04.003
- Aug 1, 2025
- Clinical lymphoma, myeloma & leukemia
Stem Cell Mobilization Yields with Daratumumab (Dara) and Lenalidomide (Len)-Containing Quadruplet Induction Therapy in Patients with Newly Diagnosed Multiple Myeloma (NDMM): A Real-World Experience at 2 Institutes.
- Research Article
12
- 10.1038/sj.bmt.1703126
- Jul 1, 2001
- Bone marrow transplantation
Forty-one patients with multiple myeloma were treated with a novel stem cell mobilisation regimen. The primary end points were adequate stem cell mobilising ability (>1% circulating CD34-positive cells) and collection (> or = 4 x 10(6) CD34-positive cells/kg), and safety. The secondary end point was activity against myeloma. The regimen (d-TEC) consisted of dexamethasone, paclitaxel 200 mg/m(2) i.v., etoposide 60 mg/kg i.v., cyclophosphamide 3 g/m(2) i.v., and G-CSF 5-10 microg/kg/day i.v. A total of 84 cycles were administered to these 41 individuals. Patient characteristics included a median age of 53 years, a median of five prior chemotherapy cycles, and a median interval of 10 months from diagnosis of myeloma to first cycle of d-TEC. Seventy-five percent of the patients had stage II or III disease, 50% had received carmustine and/or melphalan previously, and 25% had received prior radiation therapy. Eighty-eight percent of patients mobilised adequately after the first cycle of d-TEC and 91% mobilized adequately after the second cycle. An adequate number of stem cells were collected in 32 patients. Of the remaining nine patients, three mobilised, but stem cells were not collected, two mobilised but stem cell collection was < 4 x 10(6) CD34-positive cells/kg, three did not mobilise, and one died of disease progression. Major toxicities included pancytopenia, alopecia, fever and stomatitis. One patient died from multi-organ failure and progressive disease. Fifty percent of evaluable patients demonstrated a partial response and 28.6% of patients had a minor response. This novel dose-intense regimen was safe, capable of stem cell mobilisation and collection, even in heavily pre-treated patients, and active against the underlying myeloma.
- Abstract
8
- 10.1182/blood-2021-149499
- Nov 5, 2021
- Blood
Impact of Daratumumab-Containing Induction on Stem Cell Mobilization and Collection, Engraftment and Hospitalization Parameters Among Multiple Myeloma Patients Undergoing Autologous Stem Cell Transplantation
- Abstract
- 10.1182/blood-2018-99-118094
- Nov 29, 2018
- Blood
Kinetics of Myeloid-Derived Suppressor Cells during Stem Cell Mobilization and Autologous Hematopoietic Stem Cell Transplantation in Multiple Myeloma and Lymphoma Patients
- Abstract
1
- 10.1182/blood.v118.21.1934.1934
- Nov 18, 2011
- Blood
Nicotine Use During Mobilization Is Associated with More Efficient Stem Cell Collection
- Abstract
- 10.1182/blood.v104.11.1872.1872
- Nov 16, 2004
- Blood
Utility of Prophylactic Autologous Stem Cell Collection for Patients with Acute Myeloid Leukemia in First Remission.
- Abstract
- 10.1182/blood.v114.22.2154.2154
- Nov 20, 2009
- Blood
Stem Cell Mobilization Capacity Is Not a Predictor of Survival in Multiple Myeloma (MM) After Autologous Stem Cell Transplantation (ASCT).
- Abstract
4
- 10.1182/blood-2018-99-114371
- Nov 29, 2018
- Blood
Bendamustine Adversely Affects Stem Cell Mobilization Among Patients with Mantle Cell Lymphoma (MCL): A Comparison of the BR Vs RCHOP Eras in British Columbia (BC), Canada
- Abstract
3
- 10.1182/blood-2022-160071
- Nov 15, 2022
- Blood
Stem Cell Mobilization Characteristics for Transplant Eligible Patients with Newly Diagnosed Multiple Myeloma (NDMM) Treated with Carfilzomib, Lenalidomide, Dexamethasone, and Daratumumab (KRd-Dara)
- Abstract
- 10.1182/blood.v116.21.2253.2253
- Nov 19, 2010
- Blood
Peripheral Blood Stem Cell Collection In Patients Undergoing Induction Therapy with Lenalidomide Based Regimens: Failure Rates and Salvage Approaches