scholarly journals Better Transplant Outcomes with Umbilical Cord Stem Cells in Patients with Dyskeratosis Congenita

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3592-3592 ◽  
Author(s):  
Ashish O. Gupta ◽  
Steven L Shein ◽  
Jignesh D Dalal
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Umbilical Cord ◽  
Patient Population ◽  
Stem Cell Transplant ◽  
Unknown Etiology ◽  
Cell Transplant ◽  
Transplant Outcomes ◽  
Cell Transplants ◽  
Stem Cell Transplants

Abstract Background:Dyskeratosis congenita (DKC) is a rare genetic bone marrow failure (BMF) syndrome of unknown etiology with a multisystem involvement and predisposition for cancer. It presents as a classic triad of dystrophic nails, oral leukoplakia and skin hyperpigmentation. Hematopoietic stem cell transplant (HSCT) has been tried as a treatment option but there is limited data on outcomes in these patients. Methods:As the disease is rare with high mortality, there have been no prospective studies. Retrospective database analysis is an effective means to evaluate outcomes in this patient population. Our study utilized Pediatric Health Information Systems (PHIS) database, which is an administrative quality-controlled database from 43 not-for-profit children's hospital, to analyze healthcare outcomes in pediatric patients with DKC. ICD-9 code 757.39 was used to identify the disease classification and as the only condition with an indication for stem cell transplant, we isolated the cases that underwent HSCT with that diagnostic code. Results: A total of 40 patients with DKC were identified who underwent transplant. A higher incidence was noted in Caucasian males with no significant difference in mortality across gender and ethnicity. The median length of hospitalization was 48 days (range 8.5 to 160.5 days) with a mean age of hospital admission at 93 months (95% CI: 70, 117). All the transplants were allogeneic with almost 20% umbilical cord (UCT) stem cell transplants. There was 15% mortality noted in this patient population, with all of deaths occurring prior to 2012. The complication rate (including acute and chronic GVHD as well as graft failure) was noted to be 37.5%, with acute GVHD being most common (20%). Fludarabine, Alemtuzumab, Cyclophosphamide, and Melphalan based conditioning regimen were most commonly used. Cyclosporine (65%), steroids (80%), Mycophenolate mofetil (50%) and Tacrolimus (40%) were commonly used for GVHD prophylaxis. Despite small numbers, significantly lower mortality was noted in UCT as compared to the other allogeneic stem cell transplants (p<0.01). Conclusion: Limited literature is available on pediatric transplant outcome data in DKC. UCT has better transplant outcomes in DKC patients due to longer telomere lengths of donor stem cells. Larger studies are needed for to compare transplant outcomes from different sources. Disclosures No relevant conflicts of interest to declare.

Hematopoietic Stem cell transplant in National Oncology Cancer Institute of Panama. Ten Years of Experience

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4524-4524
Author(s):  
Jose Luis Franceschi Diaz
Keyword(s):  
Overall Survival ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Cell Transplants ◽  
Stem Cell Transplants ◽  
Hematopoietic Stem Cell Transplants

Abstract Abstract 4524 Panama has a population of 3.5 million inhabitants. Between 2,000 and 2,010 there were 123 hematopoietic stem cell transplants at the INSTITUTO NACIONAL DE ONCOLOGIA (ION). Even though, there are two other specialized centers doing the same procedures that we do, the ION has the greatest experience in the whole country. The objective of this document is to show you our experience and the results of a third level cancer center in an undeveloped country with limited financial resources however with pathologies and diseases that request bone marrow transplant programs installed and developed. The bone marrow transplant program (BMT) of ION initiated at the beginning of 2,000 and since then, it has been doing approximately 10–12 HSCT annually. Material and Methods: We did a review of the data during this first decade and the most important epidemiological and statistics data are presented next. Results: There were 85 autologous and 28 allogeneic hematopoietic stem cell transplants. In the allo group there were 4 reduced intensity included. In every single auto hematopoietic stem cell transplants the source of progenitor stem cells were peripheral blood and in the case of allo, hematopoietic stem cell transplants the source was the bone marrow in 18 cases and the peripheral blood in 20 cases. The mean of CD34 + progenitor cell infused was 3.23×106 (range 2.12–9.93). The peripheral blood progenitor cells were cryopreserved in liquid nitrogen until −136° celsius degrees using controlled rate temperature computerized freezers. The cells were protected from damages during the cryopreservation with cryoprotectant, in this case dimethil sulphoxide 10% was used. The age range was from 17 to 65 years old with a mean of 39 years old with a median of 40 years old. There were 66 males and 57 females. The main indication for Hematopoietic stem cell transplant was Hodgkin disease in 20 cases; follicular lymphoma in 13 cases; large cell lymphoma in 12; chronic myeloid leukemia in 21; adult acute lymphoblastic leukemia 17; adult acute myeloid leukemia in 10; multiple myeloma in 22 (2 pts. were transplanted twice upfront); severe aplastic anemia in 1; myelodisplastic syndrome in 1; lymphoblastic lymphoma in 4, and gray zone lymphoma in 1. The day 100, mortality rate for all patients was 16.3 per 100 with a range between 5–21.1 per 100 according to different diseases and types of transplants. The overall survival for all patients has a median of 59 months but when it is analyzed according to the transplant type with log rank test, there was no significative difference in the overall survival (p = 0.063). However, when all the population is grouped in categories according to diseases in Hodgkin disease; lymphomas; multiple myeloma and leukemias there is a clear difference in plot overall survival, in all four groups statistically significative with the most desfavorably outcome in the leukemia group. Indeed, if all the group is divided in two groups leukemia vs. others, there would be a clear difference in overall survival with a median overall survival of 12 months in the leukemia group that hasn’t been reached in the other group after more than 5 years of follow up (log rank p = 0.014). In all the groups there have been 55 deaths; 20 in the allo hematopoietic stem cell transplant group; and in these patients the main causes were relapse in 12 patients; graft vs. host disease in 3; infection in 4, organ toxicity in 1. There were 35 deaths in were in the auto hematopoietic stem cell transplant group, and in these patients the main group of deaths were relapse in 25 patients; infections in 3; organ toxicity in 2. Acute graft vs host disease (aGVHD). in allo hematopoietic stem cell transplant did affected desfavorably the outcome as we can see in the overall survival plot in comparison to patients without aGVHD (log rank test, p = 0.014). Chronic graft versus host disease (cGVHD) also affected desfavorably the outcome in allo hematopoietic stem cell transplant in comparison to patients with cGVHD (log rank p = 0.002). Conclusion: these results show that a BMT program can be performed in an undeveloped country with efficiency and efficacy. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Myasthenia Gravis Presenting as Graft versus Host Disease after Allogeneic Blood Stem Cell Transplant

2018 ◽  
Vol 2018 ◽  
pp. 1-3
Author(s):  
Zarir Ahmed ◽  
Martin Schoen ◽  
Nabeel Rajeh
Keyword(s):  
Stem Cell ◽  
Myasthenia Gravis ◽  
Graft Versus Host Disease ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Host Disease ◽  
Graft Versus Host ◽  
Rare Manifestation ◽  
Cell Transplants ◽  
New Onset

Myasthenia gravis is a very rare manifestation of graft versus host disease after stem cell transplants. Herein, we describe a case of new-onset myasthenia gravis after a stem cell transplant 34 months ago in a patient with myelodysplastic syndrome.

Platelet Release Occurs in the Bone Marrow Sinusoids and Spleen but Not in the Lungs Following Hematopoietic Stem Cell Transplant in the Mouse.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1710-1710
Author(s):  
Deanna Kreinest ◽  
Martha Sola ◽  
Xiao-Miao Li ◽  
Ronald Sanders ◽  
Marda Jorgensen ◽  
...  
Keyword(s):  
Stem Cells ◽  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem Cell Transplant ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Platelet Recovery ◽  
Platelet Release

Abstract The steps that lead to platelet production are poorly understood. Current theories suggest that megakaryocytes mature under the influence of contact with sinusoidal endothelium, and release platelets either in the sinusoids or in the lungs. We hypothesized that platelet release would be accentuated following hematopoietic stem cell transplant, and that sites of platelet release would be apparent during the period of platelet recovery. We transplanted highly purified hematopoietic stem cells based on lack of expression of markers for mature lineages (Linneg) and expression of Sca-1, c-kit, and Thy-1.1 (KTSL cells), and subfractionated these cells based on low expression of Rhodamine 1-2-3, into lethally irradiated hosts expressing an allelic version of glucose phosphate isomerase to identify donor and host-derived platelets. We collected bones, lungs, livers and spleens on day 7, 14, 21, and 28 post-tranplant, and stained formalin/fixed tissue with anti-Von Willebrand Factor antibody to identify megakaryocytes (5–10 animals per cohort, 2 separate experiments). We scored megakaryocytes based on their location relative to endothelial cells, and whether they were releasing platelets based on extension of proplatelet processes into the vascular spaces. Almost every megakaryocyte was associated with the endothelium during the period of platelet recovery, and we did not identify megakaryocytes that were migrating to the endothelium. We saw numerous megakaryocyte releasing platelets in both the bone marrow and the spleen during the time of platelet recovery, which occurred on days 13–28 following transplant of purified stem cells. Some of these megakaryocytes had disrupted the endothelium and were incorporated into the sinusoidal wall. Others were completely within the sinusoidal spaces. Between 30 and 50% of megakaryocytes were releasing platelets in the spleen and bone marrow at any given time following transplant, and platelet release did not correlate with the platelet counts. These levels were similar to levels of platelet release seen in healthy control mice. In contrast, we saw no identifiable megakaryocytes in the liver and lung during the period of platelet recovery. Our results suggest that in the mouse, the bone marrow and spleen, and not the lung, are major sites of platelet release following stem cell transplant.

Efficacy of Performing a Second Non-Myeloablative Allogenic Stem Cell Transplant.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3664-3664
Author(s):  
Brian J. Byrne ◽  
Joseph H. Antin ◽  
Edwin P. Alyea ◽  
Nelson J. Choa ◽  
David A. Rizzieri
Keyword(s):  
Stem Cell ◽  
Umbilical Cord ◽  
Graft Failure ◽  
Viral Disease ◽  
Cell Transplant ◽  
Allogeneic Transplant ◽  
Cell Transplants ◽  
Long Term Follow Up ◽  
Allogenic Stem Cell Transplant

Abstract Introduction: Non-myeloablative stem cell transplants are being performed for a variety of benign and malignant hematologic conditions. The reported graft failure rates have been as high as 20%. Little is reported on the safety and efficacy of a second attempt at a nonmyeloablative allogeneic transplant. Methods: Patients who underwent at least 2 nonmyeloablative allogenic transplants at either Duke University or Dana Farber Cancer Centers were included. Results: 9 patients were identified who underwent at least 2 preparatory regimens with stem cell infusion. 7 patients underwent related donor stem cell infusions and 2 patients had an umbilical cord donor. 4 patients, including the 2 with an umbilical cord donor, had primary graft failure. The remaining had secondary graft failure within 1–9 months from viral disease/therapy or disease progression. 5 of 9 received a 5 day regimen of fludarabine 125mg/m2, campath100mg and cyclophosphomide 2 gm/m2 total for a secondary preparatory regimen (FCC). Two patients received a one-day prep with fludarabine 30mg/m2, 200cGyTBI, cyclophosphamide 2gm/m2 and campath 20mg (FCC/TBI). The two umbilical cord patients received a one day prep that included TBI, cyclophosphamide 500mg/m2, fludarabine 30 mg/m2, and campath 30 mg (FCC/One). One patient with refractory CML failed to engraft, though 8/9 did (1 required a 3rd attempt), including 4 patients who had the same donor as their first transplant. Long-term follow-up revealed the graft was intact in 5 patients at 6, 7, 8, 21, 39, and 63 months. Three patients died. Conclusion: Patients who have graft failure can be transplanted with a second nonmyeloablative allotransplant. Response and long-term engraftment is possible, even using the same donor. Table 1: Patients who have undergone greater than one mini allogeneic transplant. Disease Prep #1 Donor Engraftment Prep #2 Donor Engraftment Response Key: FCC: Fludarabine, campath, cyclophosphamide; ATG FM: Fludarabine, melphalan, ATG FCC/TBI: TBI, fludarabine, campath, cyclophosphamide, FCC/One: TBI, fludarabine, cyclophosphamide, campath Thalaseemia FCC 6/6 Yes FCC 6/6 RFLP 75% CR Aplastic anemia FCC 4/6 No FCC/TBI same RFLP 99% CR Myelofibrosis FCC/TBI 6/6 RFLP 63% FCC same RFLP 98% CR CML FCC 4/6 RFLP 61% FCC 4/6 No PD Renal Cell FCC 3/6 RFLP 30% FCC same RFLP 94% PD AML/MDS FCC 4/6 RFLP 5% FCC/TBI same RFLP 89% PR AML FCC/TBI 3/6 RFLP 44% FCC 3/6 RFLP 98% CR AA/MDS ATG,FM UCB 4/6 No FCC/One UCB 4/6 RFLP 100% CR AA/MDS ATG,FM UCB 4/7 No FCC/One UCB 4/7 RFLP 100% CR

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