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Efficacy and safety of ruxolitinib in ineffective erythropoiesis suppression as a pre-transplantation treatment for pediatric patients with beta-thalassemia major
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  • Galina Ovsyannikova,
  • Dmitry Balashov,
  • Irina Demina,
  • Larisa Shelikhova,
  • Alexey Pshonkin,
  • Michael Maschan,
  • Galina Novichkova,
  • Alexei Maschan,
  • Nataliya Smetanina
Galina Ovsyannikova
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Dmitry Balashov
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Irina Demina
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Larisa Shelikhova
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Alexey Pshonkin
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Michael Maschan
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Galina Novichkova
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Alexei Maschan
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Nataliya Smetanina
Dmitry Rogachev National Medical Research Center of Pediatric Hematology Oncology and Immunology
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Abstract

Background: Ineffective erythropoiesis (IE) is the most prominent feature of transfusion-dependent beta-thalassemia (TDT), which leads to extramedullary hemopoiesis. The rejection rate in allogeneic hematopoietic stem cell transplantation (HSCT) is clearly superior in heavily transfused patients (pts) with TDT accompanied by prominent IE. Therefore, a pre-transplantation treatment bridging to HSCT is often used to reduce allosensibilization and IE. Ruxolitinib (RUX) is a JAK-1/JAK-2-inhibitor and has showed its efficacy to suppress IE and the immune system. A previously published study on RUX in adult pts with TDT has revealed that this treatment significantly reduces spleen size and is well tolerated. Procedure: Ten pts (5-14 y.o.) with TDT and an enlarged spleen were enrolled. The dose of RUX was adjusted for age: for pts younger < 11 years: 40 - 100 mg/m2 and for pts >11 years: 20 - 30 mg/m2. HSCT was performed in 8 out of the 10 pts. Results: After the first 3 months of RUX therapy the spleen volume decreased in 9 out of the 10 cases by 9.1 – 67.5% (M = 35.4%) compared to the initial size (р = 0.003). The adverse events of RUX included infectious complications, moderate thrombocytopenia as well as headache and were successfully managed by reducing the dose. The outcomes of HSCT were favorable in 7 out of the 8 cases. Conclusion: RUX is promising as a short-term pre-HSCT treatment for pediatric pts with TDT and pronounced IE.

Peer review status:ACCEPTED

23 May 2021Submission Checks Completed
23 May 2021Assigned to Editor
23 May 2021Submitted to Pediatric Blood & Cancer
25 May 2021Reviewer(s) Assigned
10 Jun 2021Review(s) Completed, Editorial Evaluation Pending
12 Jun 2021Editorial Decision: Revise Major
25 Jul 20211st Revision Received
25 Jul 2021Assigned to Editor
25 Jul 2021Submission Checks Completed
28 Jul 2021Reviewer(s) Assigned
03 Aug 2021Review(s) Completed, Editorial Evaluation Pending
03 Aug 2021Editorial Decision: Revise Minor
22 Aug 20212nd Revision Received
22 Aug 2021Submission Checks Completed
22 Aug 2021Assigned to Editor
24 Aug 2021Review(s) Completed, Editorial Evaluation Pending
24 Aug 2021Editorial Decision: Accept