Indications for Allogeneic Hematopoietic Cell Transplantation in Myelodysplastic Syndrome

Danielson, Nathalie; Byrne, Michael

doi:10.1007/s11899-019-00551-7

Cited by 6 publications

(3 citation statements)

References 75 publications

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“…Currently, several treatment options are available for patients with MDS: hypomethylating agents ( e.g ., azacytidine, decitabine,), hematopoietic stimulating agents (e.g., erythropoietin, G-CSF, luspatercept), supportive care ( e.g ., blood and platelet transfusions, antibiotics), immunomodulatory agents ( e.g ., lenalidomide, cyclosporine, thalidomide), low-dose or intensive chemotherapy, iron overload chelation (deferasirox), and hematopoietic stem cell transplantation (HSCT) [ 10 , 11 ]. The latter represents the only potentially curative option [ 12 ].…”

Section: Myelodysplastic Syndromes and Treatment Optionsmentioning

confidence: 99%

Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials

Giesinger¹,

Nasa²,

Sparano³

et al. 2021

CPEMH

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Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and blood cytopenia with a variable risk of progression to acute myeloid leukemia. The main goal of therapy for the large majority of patients is to improve health-related quality of life (HRQoL). Its rigorous assessment is now recommended in international MDS guidelines. Our review provides an overview of HRQoL results from randomized controlled trials (RCTs) in MDS patients. The literature search undertaken in PubMed identified 10 RCTs with HRQoL endpoints (all secondary) published between August 2008 and September 2020. These RCTs have helped to better understand the impact of therapies from the patient perspective and have generated valuable information that can be used to further support clinical decisions. However, the number of RCTs in MDS patients, including HRQoL endpoints, is still low. Given the importance of symptom relief and HRQoL improvement in the treatment of MDS patients, the assessment of the patient perspective in future RCTs is highly recommended to keep expanding the knowledge of the impact of new MDS therapies.

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Section: Myelodysplastic Syndromes and Treatment Optionsmentioning

confidence: 99%

Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials

Giesinger¹,

Nasa²,

Sparano³

et al. 2021

CPEMH

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“…Patients with an increased marrow blast ≥ 10% should receive pre-transplant cytoreductive treatment (intensive chemotherapy or hypomethylating agents) [ 4 ]. Despite some advances in transplant strategies over the past decades, allo-SCT remains a high-risk procedure associated with transplant-related complications and long-term survival rate of about 30–50% [ 2 , 5 ]. In this report, we present our data on the impact of patient- and disease-related factors on the outcome of allo-SCT in patients with MDS.…”

Section: Introductionmentioning

confidence: 99%

Room for Improvement: A 20-Year Single Center Experience with Allogeneic Stem Cell Transplantation for Myelodysplastic Syndromes

Duda

Wieczorkiewicz-Kabut

Spałek

et al. 2021

Indian J Hematol Blood Transfus

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Allogeneic stem cell transplantation (allo-SCT) remains the only curative therapeutic approach for patients with myelodysplastic syndromes (MDS). The aim of the study was to assess the efficacy/safety of allo-SCT as well as to identify factors influencing post-transplant survival. One hundred and two MDS patients (median age: 48 years; 57 males) who underwent allo-SCT were retrospectively evaluated. Twenty seven patients were transplanted from HLA-matched sibling and 75 patients received grafts from unrelated donors. Peripheral blood was a source of stem cell for 79 patients. Reduced intensity conditioning was used in 64 subjects. Acute and chronic graft versus host disease (GvHD) developed in 61 and 19 of patients, respectively. In total, 61 patients have died. The causes of deaths included infectious complications (n = 30), steroid-resistant GvHD (n = 17), MDS relapse (n = 9) and transformation to AML (n = 5). Non-relapse mortality and cumulative incidence of relapse at 2 years were 49.8% and 9%, respectively. 41 patients are alive at last contact and present full donor chimerism. 38 patients remain in complete hematological remission (CHR), 3 patients had CHR with incomplete platelet recovery. Median follow-up from diagnosis of MDS and transplantation are 27.1 months and 7 months respectively. Overall survival and relapse-free survival were 41% at 2 years. Increased serum ferritin level > 1000 ng/ml, presence of acute GvHD, grades III–IV acute GvHD and high hematopoietic cell transplantation-comorbidity index were found to negatively influenced survival. Allo-SCT for MDS is feasible procedure with a proportion of patients to be cured.

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“…4 Allo-HCT is also considered in patients with poor-risk myelodysplastic syndrome (MDS), myeloproliferative neoplasms, and other myeloid and lymphoid malignancies. 5,6 While durable disease control is achieved in many patients, disease relapse is a common cause of failure after HCT. Novel therapies have expanded the treatment armamentarium in both the upfront and relapsed/refractory (R/R) setting, and several of these are now used as maintenance therapies to maintain disease control after HCT.…”

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confidence: 99%

Maintenance Strategies After Hematopoietic Cell Transplantation

2020

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Hematopoietic cell transplantation (HCT) is an essential component of potentially curative therapy for patients with hematologic malignancies. High‐dose chemotherapy with autologous (auto) stem cell rescue is used to overcome chemoresistance in multiple myeloma, non‐Hodgkin lymphoma, and Hodgkin lymphoma. Alternatively, poor‐risk acute leukemias rely on the graft versus leukemia effect of allogeneic (allo) products. Long‐term remissions are feasible with both auto‐ and allo‐HCT; however, disease relapse is the leading cause of death after HCT for many patients. In recognition of this, novel therapies are being investigated in the upfront, relapsed/refractory, and post‐HCT maintenance settings to deepen response and maintain disease control. To date, the most robust data to support this approach are in multiple myeloma, where post‐transplant maintenance therapy has improved clinical outcomes. In Hodgkin lymphoma, patients with high‐risk features may benefit from post–auto‐HCT vedotin (BV) regardless of pre‐HCT BV exposure. Apart from mantle cell lymphoma, where rituximab maintenance is generally accepted, post–auto‐HCT maintenance in other forms of NHL is less established. In patients who undergo allo‐HCT, the utilization of maintenance therapy is an important component of improving post‐HCT outcomes, however, an individualized approach that considers patient factors such as residual toxicity from HCT, an immature graft with poor graft function, infection, and graft‐versus‐host disease create a complex environment for aggressive interventions. Initiation of directed agents in patients with identified mutations prior to allo‐HCT, including FLT3 in acute myeloid leukemia and Philadelphia chromosome in acute lymphoid leukemia have generally improved post‐HCT outcomes. Ongoing studies are exploring the safety and efficacy of additional maintenance strategies post–allo‐HCT in an effort to further improve post‐HCT outcomes.

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Indications for Allogeneic Hematopoietic Cell Transplantation in Myelodysplastic Syndrome

Cited by 6 publications

References 75 publications

Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials

Health-Related Quality of Life Assessment in Patients with Myelodysplastic Syndromes: Evidence from Randomized Clinical Trials

Room for Improvement: A 20-Year Single Center Experience with Allogeneic Stem Cell Transplantation for Myelodysplastic Syndromes

Maintenance Strategies After Hematopoietic Cell Transplantation

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