Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders

Mahmoud, Hossam K.; Elhaddad, Alaa; Fahmy, Omar; Samra, Mohamed; Abdelfattah, Raafat; Elnahass, Yasser; Fathy, Gamal; Abdelhady, Mohamed

doi:10.1016/j.jare.2014.11.001

Cited by 37 publications

(21 citation statements)

References 29 publications

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“…However, this kind of transplantation may be followed by many 2 of 16 complications for example, graft failure, GvHD and osteonecrosis. Graft failure in non-malignant hematological diseases as SCD, thalassemia and SCN varies significantly from 2.3-52% according to different transplant settings [2][3][4][5]. Mesenchymal stem cells (MSCs) as an essential HSC niche component [6] and as good producers of hematopoietic growth factors [7,8] are an attractive therapeutic tool to enhance engraftment in malignancies [9,10] and non-malignant hematological disorders undergoing haplo-HSCT [11].…”

Section: Introductionmentioning

confidence: 99%

“…In addition, approximately 40% of transplanted patients with hematopoietic cells of HLA-identical siblings develop GvHD compared to 59% of patients transplanted with the hematopoietic cells of unrelated donors [12]. About 29-42% of patients with SCD, thalassemia and SCN depending on the patients' age and source of hematopoietic stem cells develop GvHD after transplantation [2][3][4][5]. As MSCs express either constitutively or after their activation immunosuppressive molecules such as PGE 2 , IDO, HGF and TGF-β 1 [13,14] they are used as a second-line therapy in the treatment of steroid-refractory acute GvHD in both children and adults [15].…”

Section: Introductionmentioning

confidence: 99%

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The Phenotype and Functional Activity of Mesenchymal Stromal Cells in Pediatric Patients with Non-Malignant Hematological Diseases

Kuçi

Jordan

Wehner

et al. 2020

Cells

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As the biology of mesenchymal stromal cells (MSCs) in patients with non-malignant hematological diseases (NMHD) is poorly understood, in the current study we performed a basic characterization of the phenotype and functional activity of NMHD-MSCs. Bone marrow (BM) of patients with thalassemia major (TM) possessed a significantly higher number of nucleated cells (BM-MNCs)/mL BM than healthy donors (P < 0.0001), which however did not result in a higher number of colony forming units-fibroblast (CFU-F) per milliliter BM. In contrast, from 1 × 106 BM-MNCs of patients with sickle cell disease (SCD) were generated significantly more CFU-Fs than from TM-BM-MNCs (P < 0.013) and control group (P < 0.02). In addition, NMHD-MSCs expressed significantly lower levels of CD146 molecule, demonstrated an equal proliferation potential and differentiated along three lineages (osteoblasts, chondrocytes and adipocytes) as healthy donors’ MSCs, with exception of TM-MSCs which differentiated weakly in adipocytes. In contrast to other NMHD-MSCs and healthy donors’ MSCs, TM-MSCs demonstrated an impaired in vitro immunosuppressive potential, either. Noteworthy, the majority of the immunosuppressive effect of NMHD-MSCs was mediated through prostaglandin-E2 (PGE2), because indomethacin (an inhibitor of PGE2 synthesis) was able to significantly reverse this effect. Our results indicate therefore that NMHD-MSCs, except TM-MSCs, may be used as an autologous cell-based therapy for post-transplant complications such as graft failure, graft-versus-host disease (GvHD) and osteonecrosis.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

The Phenotype and Functional Activity of Mesenchymal Stromal Cells in Pediatric Patients with Non-Malignant Hematological Diseases

Kuçi

Jordan

Wehner

et al. 2020

Cells

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“…HSCT [17]. The regimen was well tolerated and was associated with a low incidence of complications including GVHD.…”

Section: Discussionmentioning

confidence: 99%

“…Acute and chronic GVHD were graded according to standard criteria (17).The incidence of acute GVHD was 12.6% (n=8) and…”

Section: Gvhdmentioning

confidence: 99%

ATG Based Conditioning Regimen in Stem Cells Transplantation of Fanconi Anemia: A Single Center Experience of 63 Patients

Fathy¹,

Elhaddad²,

Hk³

et al. 2017

Ann Bone Marrow Res

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Introduction: Fanconi's anemia (FA) is a rare genetic disorder. Patients with this disorder have progressive bone marrow failure, congenital abnormalities and are vulnerable to malignancy. Aim: Explaining our single center experience regarding ATG based conditioning regimen that's used for the transplantation of patients with Fanconi's Anemia. Materials and methods: Sixty-three patients with Fanconi anemia (FA) underwent stem cells transplantation from matched siblings, either from peripheral blood (PBSCsT) in fi fty nine patients , or bone marrow in four patients, according to donors' age and/weight. Mean age at BMT was 11.2 years. Conditioning regimen consisted of low-dose cyclophosphamide 5 mg/kg/d from d-5 to d-2 , fl udarabin 120mg/m2 from d-10 to d-6, CSA: 3mg/ kg/d start from d-1 and Antithymocyte globulin (ATG) was added in the pre-transplant as well as the post-transplant period at a total dose of 30 mg/kg as follows: 5mg/ kg/d (from d-4to d-1), 2.5mg/kg (d+1,d+3, d+6, d+11). We divided our patients into two cohorts based on age. The fi rst cohort aged 12 years and older and the second cohort included patients younger than 12 years old. We also studied the overall survival according to the source of stem cells (Bone Marrow vs. Peripheral blood stem cells). Results: Engraftment has been achieved in 49 (77%) patients with a mean time of 13.2 days (range, 8-26 days) SD= 3.8. The mean time for a self-sustaining platelet count of > 25 ×109/l was 14.6 days (range, 8-30 days) SD= 4.5. Eight (12.7 %) patients developed acute GVHD; none developed extensive chronic GVHD, 4 Patients developed only limited chronic GVHD. Fourteen patients died before engraftment. Conclusion: Our current results underline the importance of using ATG in the conditioning regimen for the transplantation of patients with Fanconi's anemia.

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“…Traditionally, allo-HSCT requires HLA-locus matched donors, which could be the recipient's sibling or an unrelated volunteer. The outcome of allo-HSCT is best when it is performed using an HLA-identical sibling donor [34]. However, due to incomplete donor availability, allo-HSCT from a matched sibling donor is available for only 30% of patients [567]; for the remaining 70%, the next-best option is to receive allo-HSCT from an unrelated HLA-matched donor.…”

Section: Introductionmentioning

confidence: 99%

Physicians' preferences and perceptions regarding donor selection in allogeneic stem cell transplantation in Korea when a matched domestic donor is not available

Shin

Lee

et al. 2017

Blood Res

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BackgroundA number of alternative donor options exist for patients who fail to find domestic HLA-matched donors for allogeneic hematopoietic stem cell transplantation (allo-HSCT). We assessed physicians' perspectives on allo-HSCT donor selection when a matched domestic donor is not available.MethodsWe administered a questionnaire survey to 55 hematologists (response rate: 28%) who attended the annual spring conference of the Korean Society of Haematology in 2015. The questionnaire contained four clinical allo-HSCT scenarios and the respondents were asked to choose the most preferred donor among the given options.ResultsIn all four scenarios, the hematologists preferred a matched international donor over partially mismatched unrelated domestic or haplo-matched family donors. The numbers of hematologists who chose a matched international donor (HLA 8/8) in cases of acute myeloid leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia, and aplastic anemia were 37 (67.3%), 41 (74.6%), 33 (60.0%), and 36 (65.5%), respectively. The important factors that affected donor selection included “expecting better clinical outcomes (40.5%)” and “lower risk of side effects (23.4%).” The majority of participants (80%) responded that allo-HSCT guidelines for donor selection customized for the Korean setting are necessary.ConclusionAlthough hematologists still prefer perfectly matched foreign donors when a fully matched domestic allo-HSCT donor is not available, we confirmed that there was variation in their responses. For evidence-based clinical practice, it is necessary to provide further comparative clinical evidence on allo-HSCT from haplo-matched family donors and fully matched unrelated international donors.

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Allogeneic hematopoietic stem cell transplantation for non-malignant hematological disorders

Cited by 37 publications

References 29 publications

The Phenotype and Functional Activity of Mesenchymal Stromal Cells in Pediatric Patients with Non-Malignant Hematological Diseases

The Phenotype and Functional Activity of Mesenchymal Stromal Cells in Pediatric Patients with Non-Malignant Hematological Diseases

ATG Based Conditioning Regimen in Stem Cells Transplantation of Fanconi Anemia: A Single Center Experience of 63 Patients

Physicians' preferences and perceptions regarding donor selection in allogeneic stem cell transplantation in Korea when a matched domestic donor is not available

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