Transfus Apher Sci. 2014 Dec 23. S1473-0502(14)00259-6. doi:10.1016/j.transci.2014.12.021.
Successful treatment of a 3-year-old boy with hepatitis-associated aplastic anemia with combination of auto-umbilical cord blood transplantation and immunosuppressive therapy.
Liang C, Wei J, Jiang E, Ma Q, Pang A, Feng S, Han M.
Hematopoietic Stem Cell Transplantation Center, Institute of Hematology and Blood Diseases Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Tianjin 300020, China.
Viene descritto il caso di un bambino di 3 anni di età affetto da anemia aplastica associata ad epatite. E’ stato trattato con successo tramite un trapianto autologo del suo sangue di cordone ombelicale in combinazione con una terapia immunosoppressiva.
Abstract
In this work we describe a 3-year-old boy with hepatitis-associated aplastic anemia (HAAA) treated successfully with autologous cord blood transplantation combined with immunosuppressive therapy. There is little previous experience in the utility of autologous cord blood transplantation in the treatment of HAAA. Nowadays, for patients born after 1980, an HLA matched sibling donor is not usually available because of the family planning policy in our country. So more and more parents choose to preserve the umbilical cord blood for their children. We consider it a new effective choice for the treatment of HAAA, especially for the pediatric patients. Copyright © 2014 Elsevier Ltd. All rights reserved. KEYWORDS: Autologous transplantation; Cord blood; Hepatitis-associated aplastic anemia
J Pediatr. 2014 May;164(5):973-979.e1. doi: 10.1016/j.jpeds.2013.11.036. Epub 2013 Dec 31.
Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy.
Cotten CM, Murtha AP, Goldberg RN, Grotegut CA, Smith PB, Goldstein RF, Fisher KA, Gustafson KE, Waters-Pick B, Swamy GK, Rattray B, Tan S, Kurtzberg J. Duke University, Durham, NC.
OBIETTIVO: Valutare la fattibilità e la sicurezza del fornire sangue autologo del cordone ombelicale (umbilical cord blood, UCB) a neonati con encefalopatia ipossico-ischemica (HIE).
DISEGNO dello STUDIO: Sono stati arruolati bambini della Terapia Intensiva Neonatale che sono stati “raffreddati” (induzione di ipotermia) per HIE e che avevano a disposizione UCB. Sono state registrate le caratteristiche delle cellule infuse e i segni vitali pre- e post-infusione. Sono stati poi confrontati i risultati di questi bambini (mortalità, capacità di alimentarsi per via orale alla dimissione, sopravvivenza a 1 anno, capacità cognitiva, linguaggio e sviluppo motorio) con i bambini trattati solo con l’ipotermia, ovvero bambini che non avevano cellule autologhe cordonali di cui disporre.
RISULTATI: Ventitrè bambini sono stati “raffreddati” e hanno ricevuto le cellule del UCB. I segni vitali, tra cui saturazione dell’ossigeno, erano simili prima e dopo le infusioni nelle prime 48 ore dopo la nascita.
I due gruppi di neonati hanno avuto esiti simili alla dimissione dall’ospedale. Il 74% dei bambini trattati con ipotermia + cellule cordonali e il 41% di quelli trattati solo con l’ipotermia sono sopravvissuti dopo 1 anno con punteggi elevati.
CONCLUSIONI: Raccolta, preparazione e infusione di cellule fresche autologhe di UCB per l’uso nei neonati con HIE è fattibile. È necessario uno studio in doppio cieco randomizzato.
Abstract
OBJECTIVE: To assess feasibility and safety of providing autologous umbilical cord blood (UCB) cells to neonates with hypoxic-ischemic encephalopathy (HIE).
STUDY DESIGN: We enrolled infants in the intensive care nursery who were cooled for HIE and had available UCB in an open-label study of non-cyropreserved autologous volume- and red blood cell-reduced UCB cells (up to 4 doses adjusted for volume and red blood cell content, 1-5 × 10(7) cells/dose). We recorded UCB collection and cell infusion characteristics, and pre- and post-infusion vital signs. As exploratory analyses, we compared cell recipients’ hospital outcomes (mortality, oral feeds at discharge) and 1-year survival with Bayley Scales of Infant and Toddler Development, 3rd edition scores ≥85 in 3 domains (cognitive, language, and motor development) with cooled infants who did not have available cells.
RESULTS: Twenty-three infants were cooled and received cells. Median collection and infusion volumes were 36 and 4.3 mL. Vital signs including oxygen saturation were similar before and after infusions in the first 48 postnatal hours. Cell recipients and concurrent cooled infants had similar hospital outcomes. Thirteen of 18 (74%) cell recipients and 19 of 46 (41%) concurrent cooled infants with known 1-year outcomes survived with scores >85.
CONCLUSIONS: Collection, preparation, and infusion of fresh autologous UCB cells for use in infants with HIE is feasible. A randomized double-blind study is needed. Copyright © 2014 Elsevier Inc. All rights reserved.
Comment in Stem cell-based therapy for newborn lung and brain injury: feasible, safe, and the next therapeutic breakthrough? [J Pediatr. 2014]
PMID:24388332[PubMed – indexed for MEDLINE] PMCID:PMC3992180[Available on 2015/5/1]
Mymensingh Med J. 2013 Jan;22(1):210-7.
Autologous or allogenic uses of umbilical cord blood whole or RBC transfusion – a review.
1Department of Transfusion Medicine, Mymensingh Medical College, Mymensingh, Bangladesh.
Una volta il cordone ombelicale e la placenta erano considerati un rifiuto e venivano eliminati dopo il parto, ma ora il sangue del cordone è una valida fonte di trapianto di cellule staminali ematopoietiche. Neonati prematuri ad alto rischio richiedono trasfusioni di globuli rossi per l’anemia. Una proprietà unica del sangue del cordone ombelicale (CB, cord blood) è il suo alto contenuto di cellule immature progenitrici ematopoietiche. Il sangue placentare può essere raccolto sterilmente e conservato a 4 ° C. .. Le analisi chimiche e colturali indicano che il sangue placentare umano può essere raccolto in modo sicuro e conservato in modo efficace per la terapia trasfusionale allogenica o autologa. Nella pratica trasfusionale neonatale, sono stati fatti molti sforzi per fornire ai neonati prematuri globuli rossi autologhi, specialmente per quelli nati prima di 32 settimane di gestazione. In India sono state osservate trasfusioni senza effetti negativi in una vasta gamma di pazienti che ha ricevuto sangue allogenico cordonale. È stato anche proposto l’uso di CB per le piccole trasfusioni allogeniche nei bambini anemici in Africa o in aree endemiche della malaria. Uno studio ha dimostrato che donazione e trasfusione di CB sarebbero accettabili per le donne che vivono in Kenya. Nei Paesi con risorse limitate si potrebbe beneficiare maggiormente di questo prodotto trasfusionale facilmente disponibile.
Abstract
Once Umbilical Cord with Placenta considered a biological waste product and generally discarded after delivery but now cord blood has emerged as a viable source of hematopoietic stem cell transplantation. High-risk premature infants require red cell transfusions for anemia. A unique property of cord blood (CB) for its high content of immature hematopoietic progenitor cells (HPCs). Placental blood for autologous transfusions can be collected with aseptic precaution/sterilely into citrate-phosphate-dextrose and stored at 4°C. During storage for 8 days, the placental red cell content of adenosine triphosphate remained normal. The 2,3,-diphosphoglycerate concentration of cells stored beyond 8 days declined sharply. So we have to store umbilical cord blood (UCB) within 7 days for its best result. During storage, placental blood underwent an exchange of extra-cellular Na+ and K+, but no change in glutathione content. Hemolysis was less than 1 percent. Bacteriologic and fungal cultures remained sterile. These suggest that human placental blood can be collected safely and preserved effectively for autologous/allogenic transfusion therapy. In neonatal transfusion practice, efforts have been made to provide premature infants with autologous red blood cell (RBC), especially those born before 32 gestational weeks. In India no adverse transfusion effects were seen in a wide variety of patients that received (pooled) allogeneic fresh whole blood / UCB transfusions. The use of UCB for small volume allogeneic transfusions in anaemic children in Africa or in malaria endemic areas has also been proposed. A preclinical study showed that donation and transfusion of UCB would be acceptable to women living in Mombasa, Kenya. In view of the small volumes RBC per unit that can be collected, it is most likely that anaemic children need of a small volume of transfusions. In resource-restricted countries would benefit most from this easily available transfusion product. PMID:23416835[PubMed – indexed for MEDLINE]
Stem Cells Transl Med. 2015 Feb;4(2):195-206. doi: 10.5966/sctm.2014-0195. Epub 2015 Jan 5.
Safety and feasibility for pediatric cardiac regeneration using epicardial delivery of autologous umbilical cord blood-derived mononuclear cells established in a porcine model system.
Cantero Peral S, Burkhart HM, Oommen S, Yamada S, Nyberg SL, Li X, O’Leary PW, Terzic A, Cannon BC, Nelson TJ; Wanek Program Porcine Pipeline Group; Wanek Program Porcine Pipeline Group.
Collaborators: Edgerton SL, Suddendorf SH, Krage S, Rice M, Rysavy JA, Powers JM, Rasmussen BW, Miller JM, Paulson TL, Lindquist RK, Reece CL, Miller AR, Padley DJ, Wentworth MA, Greene AC, Andrews AG, Nelson TJ, O’Leary PW, Olson TM, Terzic A.
Division of General Internal Medicine, Center for Regenerative Medicine, Pediatric Cardiothoracic Surgery, Division of Cardiovascular Diseases, Transplant Center, Division of Biomedical Statistics and Informatics, Division of Pediatric Cardiology, Department of Molecular Pharmacology and Experimental Therapeutics, and Mayo Clinic, Rochester, Minnesota, USA; +Program of Doctorate of Internal Medicine, Autonomous University of Barcelona, Barcelona, Spain.
Si cercano nuove opzioni terapeutiche per migliorare i risultati a lungo termine nelle malattie cardiache congenite per le quali si interviene chirurgicamente. Non ci sono studi sugli effetti a lungo termine del trapianto di cellule autologhe in età pediatrica, nonostante sia stato dimostrato che cellule staminali inducono rigenerazione cardiaca. In questo studio sono state iniettate cellule mononucleate autologhe del cordone ombelicale nel miocardio del ventricolo destro di maialini ed è stato esaminato il rischio della manovra, in particolare in termini di aritmie e infezioni.
Abstract
Congenital heart diseases (CHDs) requiring surgical palliation mandate new treatment strategies to optimize long-term outcomes. Despite the mounting evidence of cardiac regeneration, there are no long-term safety studies of autologous cell-based transplantation in the pediatric setting. We aimed to establish a porcine pipeline to evaluate the feasibility and long-term safety of autologous umbilical cord blood mononuclear cells (UCB-MNCs) transplanted into the right ventricle (RV) of juvenile porcine hearts. Piglets were born by caesarean section to enable UCB collection. Upon meeting release criteria, 12 animals were randomized in a double-blinded fashion prior to surgical delivery of test article (n = 6) or placebo (n = 6). The UCB-MNC (3 × 10(6) cells per kilogram) or control (dimethyl sulfoxide, 10%) products were injected intramyocardially into the RV under direct visualization. The cohorts were monitored for 3 months after product delivery with assessments of cardiac performance, rhythm, and serial cardiac biochemical markers, followed by terminal necropsy. No mortalities were associated with intramyocardial delivery of UCB-MNCs or placebo. Two animals from the placebo group developed local skin infection after surgery that responded to antibiotic treatment. Electrophysiological assessments revealed no arrhythmias in either group throughout the 3-month study. Two animals in the cell-therapy group had transient, subclinical dysrhythmia in the perioperative period, likely because of an exaggerated response to anesthesia. Overall, this study demonstrated that autologous UCB-MNCs can be safely collected and surgically delivered in a pediatric setting. The safety profile establishes the foundation for cell-based therapy directed at the RV of juvenile hearts and aims to accelerate cell-based therapies toward clinical trials for CHD. ©AlphaMed Press. KEYWORDS: Autologous umbilical cord blood; Congenital heart disease; Intramyocardial delivery; Porcine; Right ventricle; Safety
Ann Plast Surg.2014;72(6):S176-83. doi: 10.1097/SAP.0000000000000107.
Osteoinduction of umbilical cord and palate periosteum-derived mesenchymal stem cells on poly(lactic-co-glycolic) acid nanomicrofibers.
Caballero M, Pappa AK, Roden KS, Krochmal DJ, van Aalst JA.
Viene illustrata una tecnica per creare tessuto osseo a partire da cellule staminali di cordone ombelicale al fine di riparare gravi difetti craniofacciali (dovuti a anomalie congenite, traumi, interventi chirurgici di asportazione di tumori).
Abstract
The need for tissue-engineered bone to treat complex craniofacial bone defects secondary to congenital anomalies, trauma, and cancer extirpation is sizeable. Traditional strategies for treatment have focused on autologous bone in younger patients and bone substitutes in older patients. However, the capacity for merging new technologies, including the creation of nanofiber and microfiber scaffolds with advances in natal sources of stem cells, is crucial to improving our treatment options. The advantages of using smaller diameter fibers for scaffolding are 2-fold: the similar fiber diameters mimic the in vivo extracellular matrix construct and smaller fibers also provide a dramatically increased surface area for cell-scaffold interactions. In this study, we compare the capacity for a polymer with Federal Drug Administration approval for use in humans, poly(lactic-co-glycolic) acid (PLGA) from Delta polymer, to support osteoinduction of mesenchymal stem cells (MSCs) harvested from the umbilical cord (UC) and palate periosteum (PP). Proliferation of both UC- and PP-derived MSCs was improved on PLGA scaffolds. The PLGA scaffolds promoted UC MSC differentiation (indicated by earlier gene expression and higher calcium deposition), but not in PP-derived MSCs. Umbilical cord-derived MSCs on the PLGA nanomicrofiber scaffolds have potential clinical utility in providing solutions for craniofacial bone defects, with the added benefit of earlier availability. PMID:24691324 [PubMed – indexed for MEDLINE] PMCID:PMC4049008[Available on 2015/6/1]