scholarly journals Prospective study on Prophylactic Micafungin sodium against Invasive Fungal Disease during Neutropenia in Pediatric & Adolescent Patients undergoing Autologous Hematopoietic Stem Cell Transplantation

2021 ◽  
Author(s):  
Bo Kyung Kim ◽  
Jung Yoon Choi ◽  
Kyung Taek Hong ◽  
Hong Yul An ◽  
Hee Young Shin ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Treatment Success ◽  
Open Label ◽  
Hematopoietic Stem ◽  
Adolescent Patients ◽  
Autologous Hsct

Background Invasive fungal diseases (IFDs) increase the mortality rate of patients with neutropenia who receive chemotherapy or have previously undergone hematopoietic stem cell transplantation (HSCT). Micafungin is a broad-spectrum echinocandin, with minimal toxicity and low drug interactions. We therefore investigated the efficacy and safety of prophylactic micafungin in pediatric and adolescent patients who underwent autologous HSCT. Methods This was a phase II, prospective, single-center, open-label, and single-arm study. From November 2011 to February 2017, 125 patients were screened from Seoul National University Children’s Hospital, Korea, and 112 were enrolled. Micafungin was administered intravenously at a dose of 1 mg/kg/day (maximum 50 mg/day) from day 8 of autologous HSCT until neutrophil engraftment. Treatment success was defined as the absence of proven, probable, or possible IFD up to 4 weeks after therapy. Results The study protocol was achieved without premature interruption in 110 patients (98.2%). The reasons interrupting micafungin treatment included early death (n=1) and patient refusal (n=1). Treatment success was achieved in 109 patients (99.1%). Only one patient was diagnosed with probable IFD. No patients were diagnosed with possible or proven IFD. In the full analysis set, 21 patients (18.8%) experienced 22 adverse events (AEs); however, all AEs were classified as “unlikely” related to micafungin. No patient experienced grade IV AEs nor discontinued treatment and none of the deaths were related to micafungin. Conclusions Our study demonstrated that micafungin is a safe and effective option for antifungal prophylaxis in pediatric patients who underwent autologous HSCT, with promising efficacy without significant AEs.

scholarly journals Human NK Cells in Autologous Hematopoietic Stem Cell Transplantation for Cancer Treatment

Cancers ◽  
2021 ◽  
Vol 13 (7) ◽  
pp. 1589
Author(s):  
Ane Orrantia ◽  
Iñigo Terrén ◽  
Gabirel Astarloa-Pando ◽  
Olatz Zenarruzabeitia ◽  
Francisco Borrego
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Nk Cells ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cell Biology ◽  
Nk Cell ◽  
Hematopoietic Stem ◽  
Autologous Hsct

Natural killer (NK) cells are phenotypically and functionally diverse lymphocytes with the ability to recognize and kill malignant cells without prior sensitization, and therefore, they have a relevant role in tumor immunosurveillance. NK cells constitute the main lymphocyte subset in peripheral blood in the first week after hematopoietic stem cell transplantation (HSCT). Although the role that NK cells play in allogenic HSCT settings has been documented for years, their significance and beneficial effects associated with the outcome after autologous HSCT are less recognized. In this review, we have summarized fundamental aspects of NK cell biology, such as, NK cell subset diversity, their effector functions, and differentiation. Moreover, we have reviewed the factors that affect autologous HSCT outcome, with particular attention to the role played by NK cells and their receptor repertoire in this regard.

Hematopoietic Stem Cell Transplantation for Autoimmune Disease

2020 ◽  
Vol 72 (1) ◽  
Author(s):  
Tobias Alexander ◽  
Raffaella Greco ◽  
John A. Snowden
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Autoimmune Diseases ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Publication Date ◽  
Hematopoietic Stem ◽  
Wide Range ◽  
Autologous Hsct

The introduction of targeted biologic therapies has changed the treatment landscape for autoimmune diseases (ADs) substantially, but although these therapies provide more specificity, they require continuous administration, rarely restore organ function or reverse disability, and are not curative. Over the last 25 years, hematopoietic stem cell transplantation (HSCT) has been increasingly used to treat patients in whom the risk:benefit ratio of HSCT is acceptable. In contrast to chronic suppression of immune function, this intensive one-off procedure aims to provide treatment-free remissions by the reinduction of self-tolerance. The European Society of Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) has been central to development of this approach, with over 3,300 HSCT registrations for ADs. Recent data have improved the evidence base to support autologous HSCT in multiple sclerosis, systemic sclerosis, and Crohn's disease, along with a wide range of rarer disease indications, and autologous HSCT has become an integral part of treatment algorithms in various ADs. Expected final online publication date for the Annual Review of Medicine, Volume 72 is January 27, 2021. Please see http://www.annualreviews.org/page/journal/pubdates for revised estimates.

Autologous Non-Myeloablative Hematopoietic Stem Cell Transplantation for Refractory Systemic Vasculitis.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5457-5457 ◽  
Author(s):  
Laisvyde Statkute ◽  
Yu Oyama ◽  
Walter G. Barr ◽  
Jolita Satkus ◽  
Yvonne Loh ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Systemic Vasculitis ◽  
Sustained Remission ◽  
Hematopoietic Stem ◽  
Autologous Hsct

Abstract Background Current mortality rate in systemic vasculitis (SV) patients with the use of cytotoxic / immunosuppressive therapy is over 20 % at 5 years in some studies. For patients refractory to conventional therapy new strategies aimed at aggressive induction of remission and relapse prevention are being sought. We here report our single center experience in treating 4 patients with refractory SV employing non-myeloablative autologous hematopoietic stem cell transplantation (HSCT). Patients and Methods Four patients with refractory SV (2 - with neurovascular Behcet’s disease, 1 - with neurovascular Sjogren’s syndrome, and 1 - with Wegener’s granulomatosis) were involved in an IRB and FDA approved phase I clinical trial of high dose chemotherapy and autologous HSCT. Peripheral blood stem cells were mobilized with cyclophosphamide (Cy) 2g/m2 IV and granulocyte-colony stimulating factor 10 mcg/kg/day SQ and were CD34+ enriched. Conditioning regimen consisted of Cy 200 mg/kg and rabbit anti-thymocyte globulin 5.5 mg/kg IV. Disease activity and cumulative organ damage were assessed using Birmingham Vasculitis Activity Score (BVAS) and Vasculitis Damage Index (VDI), respectively. Primary and secondary endpoints were transplant-related toxicity, survival and change in BVAS and VDI after the transplant. Results All 4 patients tolerated HSCT well without transplant related mortality or any significant toxicity. At median follow up of 28 (range 22–35) months all patients are alive. Three patients (1 each with Behcet’s, Sjogren’s, and Wegener’s) entered a sustained remission at 6, 6 and 24 months after the transplant. Both, BVAS and VDI, markedly decreased within 6 months post-transplant. In a patient with WG C-ANCA levels became undetectable at 24 month evaluation corresponding to clinical and radiological improvement. All 3 patients who achieved remission discontinued immunosuppressive therapy at the time of transplant and have not required since. Two patients with pre-transplant steroid dependency discontinued prednisone at 6 and 14 months after HSCT. One patient with BD who is positive HLA-B51, now 22 months since HSCT, has never achieved remission and is still requiring immunosuppressive therapy. Conclusion We suggest autologous HSCT utilizing a non-myeloablative regimen is a safe and effective treatment for select patients with SV refractory to conventional immunosuppressive therapies. However, in patients with significant genetic predisposition, more aggressive approach such as allogeneic HSCT or cord blood transplantation might be considered.

Autologous Non-Myeloablative Hematopoietic Stem Cell Transplantation for Diffuse Scleroderma.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2920-2920
Author(s):  
Yu Oyama ◽  
Laisvyde Statkute ◽  
Walter Barr ◽  
Ann Traynor ◽  
Richard Burt
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Unknown Etiology ◽  
Hematopoietic Stem ◽  
Cell Counts ◽  
Diffuse Scleroderma ◽  
Autologous Hsct

Abstract Background &Aims Scleroderma is an immune mediated vasculopathy of unknown etiology. There is no known therapy that could alter the natural course of the disease. Patients with diffuse cutaneous disease or visceral involvement has an overall 10-year survival of 35 to 68%. Autologous hematopoietic stem cell transplantation (HSCT) with myeloablative regimen has been performed, but with substantial mortality and morbidity especially pulmonary and renal toxicity. We have performed autologous HSCT utilizing non-myeloablative, but lymphoablative conditioning. Methods We conducted a phase 1 HSCT study in 9 patients with diffuse scleroderma with poor clinical features. Candidates were less than 65 years old with Rodman score of more than 14 or diffusion capacity less than 80%, interstitial lung disease, elevated ESR, renal involvement, or abnormal electrocardiogram. Patients with pulmonary hypertension (systolic pressure > 45 mm Hg) were excluded. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte colony-stimulating factor. The graft was not manipulated. The conditioning regimen consisted of 200mg/kg cyclophosphamide (CY), and 7.5mg/kg rabbit antithymocyte globulin (ATG). Results The procedure was well tolerated with anticipated cytopenias, neutropenic fever, and fluid overload that were easy to manage and without mortality. The median days for neutrophil and platelet engraftment were 8 (range 7–9) and 8 (range 0–10), respectively. The median infused CD34+ and CD3+ cell counts were 8.31 x 106/kg (range 2.35–14.7) and 2.03 x 108/kg (range 0.41–6.83), respectively. There was a marked improvement of skin score in all subjects, whereas cardiac (ejection fraction, pulmonary arterial pressure), pulmonary function (DLCO) and renal function (creatinine) remained stable. One patient with advanced disease with poor performance status died 2 years after the transplant with progressive disease. After median follow-up of 20 months (range 5–32), the overall survival is 89% (eight out of nine) and progression-free survival with continuing improvement is 67% (six out of nine. 2 patients developed recurrence of skin tightness without compromising organ function. Both of them were placed on mycophenolate mofetil with gradual improvement of skin tightness. Conclusion Autologous HSCT with CY/ATG is safe and is effective. A randomized study (ASSIST: American Scleroderma Stem Cell vs. Immune Suppression Trial), comparing HSCT to intravenous pulse cyclophosphamide is now enrolling patients. Figure Figure

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