scholarly journals Advances in the management of α-thalassemia major: reasons to be optimistic

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 592-599
Author(s):  
Paulina Horvei ◽  
Tippi MacKenzie ◽  
Sandhya Kharbanda
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Phase 1 ◽  
Globin Gene ◽  
Severe Disease ◽  
Thalassemia Major ◽  
In Utero ◽  
Number Of Children ◽  
Ongoing Phase

Abstract α-Thalassemia major (ATM) is a severe disease resulting from deletions in all 4 copies of the α-globin gene. Although it is usually fatal before birth, the advent of in utero transfusions has enabled survival of a growing number of children. Postnatal therapy consists of chronic transfusions or stem cell transplantation, similar to patients with β-thalassemia major. In this review, we discuss the experience with postnatal stem cell transplantation in patients with ATM, as well as the ongoing phase 1 clinical trial of in utero stem cell transplantation for this condition.

166Ho-DOTMP plus melphalan followed by peripheral blood stem cell transplantation in patients with multiple myeloma: results of two phase 1/2 trials

Blood ◽  
2003 ◽  
Vol 102 (7) ◽  
pp. 2684-2691 ◽  
Author(s):  
Sergio Giralt ◽  
William Bensinger ◽  
Mark Goodman ◽  
Donald Podoloff ◽  
Janet Eary ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Phase 1 ◽  
High Dose ◽  
Two Phase ◽  
Hematopoietic Stem

Abstract Holmium-166 1, 4, 7, 10-tetraazcyclododecane-1, 4, 7, 10-tetramethylenephosphonate (166Ho-DOTMP) is a radiotherapeutic that localizes specifically to the skeleton and can deliver high-dose radiation to the bone and bone marrow. In patients with multiple myeloma undergoing autologous hematopoietic stem cell transplantation two phase 1/2 dose-escalation studies of high-dose 166Ho-DOTMP plus melphalan were conducted. Patients received a 30 mCi (1.110 Gbq) tracer dose of 166Ho-DOTMP to assess skeletal uptake and to calculate a patient-specific therapeutic dose to deliver a nominal radiation dose of 20, 30, or 40 Gy to the bone marrow. A total of 83 patients received a therapeutic dose of 166Ho-DOTMP followed by autologous hematopoietic stem cell transplantation 6 to 10 days later. Of the patients, 81 had rapid and sustained hematologic recovery, and 2 died from infection before day 60. No grades 3 to 4 nonhematologic toxicities were reported within the first 60 days. There were 27 patients who experienced grades 2 to 3 hemorrhagic cystitis, only 1 of whom had received continuous bladder irrigation. There were 7 patients who experienced complications considered to be caused by severe thrombotic microangiopathy (TMA). No cases of severe TMA were reported in patients receiving in 166Ho-DOMTP doses lower than 30 Gy. Approximately 30% of patients experienced grades 2 to 4 renal toxicity, usually at doses targeting more than 40 Gy to the bone marrow. Complete remission was achieved in 29 (35%) of evaluable patients. With a minimum follow-up of 23 months, the median survival had not been reached and the median event-free survival was 22 months. 166Ho-DOTMP is a promising therapy for patients with multiple myeloma and merits further evaluation. (Blood. 2003;102:2684-2691)

Quality of life in Patients with β-thalassemia Major: Short-term and Long-term Effects after Haematopoietic Stem Cell Transplantation

2021 ◽  
Vol 16 ◽  
Author(s):  
Lu Zhai ◽  
Yuhua Liu ◽  
Rongrui Huo ◽  
Zhaofang Pan ◽  
Juan Bin ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Stem Cell ◽  
Physical Therapy ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Psychological Intervention ◽  
Thalassemia Major ◽  
Haematopoietic Stem Cell

Background: Allogeneic haematopoietic stem cell transplantation (ALLO-HSCT) is a potentially curative approach to treat β-thalassemia major (β-TM). Objective and Methods: To assess the quality of life (QOL) of patients with β-TM after ALLO-HSCT, we searched PubMed, Embase, Web of Science, and Medline for articles on the quality of life (QOL) of patients with β-TM from 1 Feb 2020 to 31 Mar 2020. Results: Our review revealed that the QOL of patients with β-TM after ALLO-HSCT from a sibling donor is higher than that of patients that received blood infusion and iron-chelating therapy. Survivors of ALLO-HSCT have a QOL as good as that of a healthy population and the ability to return to normal life. However, studies thus far are limited to investigations with a few patients with β-TM who received ALLO-HSCT of the bone marrow (BM) from a sibling donor or related donor. Graft vs. host disease, patient age, gender, sexual desire, health condition, psychological state, financial and employment stress, and social support contributed to a worse QOL after ALLO-HSCT. Medicine usage, physical therapy, and psychological intervention may help improve the decline in QOL related to ALLO-HSCT in patients with β-TM. Conclusion: Doctors and nurses must focus on implementing medicine usage, physical therapy and psychological intervention to improve the decline in QOL related to ALLO-HSCT.

scholarly journals Total body irradiation in a case of thalassemia major with source to axis distance based planning: A case report

2020 ◽  
pp. 25-28
Author(s):  
Mayuresh D. Virkar ◽  
Rajkumar Chauhan ◽  
Pranav Chadha ◽  
Kaustav Talapatra ◽  
Reuben Jake Rodrigues ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Total Body Irradiation ◽  
Conditioning Regimen ◽  
Thalassemia Major ◽  
Entire Body ◽  
Hematopoietic Stem ◽  
Total Body

Background: The use of total body radiation (TBI) before hematopoietic stem cell transplantation (HSCT) would increase the engraftment without transplant-related morbidity or mortality among Thalassemia major (TM) cases. Case presentation: A 2-year-old female child, diagnosed with TM was scheduled for haploidentical allogenic transplant-based protocol, and after that, based on protocol she was scheduled to undergo a single session of TBI as a conditioning regimen before haploidentical allogenic hematopoietic stem cell tranplant. A total dose of 4 Gy was administered.. The incidence of graft failure was reduced as TBI was used before allogeneic stem cell transplantation. TBI provided a uniform dose of radiation to the entire body, penetrating areas such as the central nervous system (CNS) and testes. Conclusion: Total Body Irradiation with the SAD technique is the most effective way of treatment. As it is comfortable for the patient to undergo, easily reproducible, and it helps to achieve a uniform dose distribution.

Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study

The Lancet ◽  
2002 ◽  
Vol 360 (9327) ◽  
pp. 130-137 ◽  
Author(s):  
Isabelle André-Schmutz ◽  
Françoise Le Deist ◽  
Salima Hacein-Bey-Abina ◽  
Ellen Vitetta ◽  
John Schindler ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Graft Versus Host Disease ◽  
Immune Reconstitution ◽  
Phase 1 ◽  
Host Disease ◽  
Graft Versus Host ◽  
Haemopoietic Stem Cell ◽  
Haemopoietic Stem Cell Transplantation
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