scholarly journals Thrombotic Thrombocytopenic Purpura in Childhood

Blood ◽  
1962 ◽  
Vol 19 (2) ◽  
pp. 181-199 ◽  
Author(s):  
JAMES B. MACWHINNEY ◽  
JAMES T. PACKER ◽  
GERALD MILLER ◽  
ROBERT M. GREENDYKE
Keyword(s):  
Red Blood Cells ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Clinical Features ◽  
Blood Cells ◽  
Patient Case ◽  
Thrombocytopenic Purpura ◽  
Renal Vessels ◽  
Kidney Involvement ◽  
Histologic Changes

Abstract (1) Two cases of thrombotic thrombocytopenic purpura (TTP) occurring in childhood are described. Case 1 is unique in that the patient survived for 12 years. (2) The clinical features of 19 reported cases of TTP in children are reviewed. (3) The presence of morphologic abnormalities of red blood cells and the regular occurrence of kidney involvement in this disorder is emphasized. In one patient (Case 2), histologic changes of the disease were limited to the kidney. (4) Certain hematologic and histologic features shared by TTP and eclampsia are described. (5) Unusual histologic lesions of renal vessels are described.

scholarly journals Thrombotic Thrombocytopenic Purpura and Sickle Cell Crisis

2008 ◽  
Vol 16 (2) ◽  
pp. 224-227 ◽  
Author(s):  
Suresh G. Shelat
Keyword(s):  
Red Blood Cells ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Sickle Cell ◽  
Blood Cells ◽  
Signs And Symptoms ◽  
Activity Level ◽  
Acute Chest Syndrome ◽  
Thrombocytopenic Purpura ◽  
Nucleated Red Blood Cells ◽  
Elevated Lactate

Described is a case of acute chest syndrome in a sickle-cell patient (hemoglobin SS) who also developed signs and symptoms of thrombotic thrombocytopenic purpura, including thrombocytopenia and hemolysis (anemia, elevated lactate dehydrogenase, presence of schistocytes, dark-colored plasma, and elevations in nucleated red blood cells). The ADAMTS13 activity level was normal. Discussed are the diagnosis and therapeutic management issues and the challenges of differentiating the vasoocclusive and hemolytic complications of sickling red blood cells from the thrombotic microangiopathy of thrombotic thrombocytopenic purpura.

scholarly journals Adamts13-Cultured Red Blood Cells to Treat Thrombotic Thrombocytopenic Purpura

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 89-89
Author(s):  
Kai Wang ◽  
Khulan Batbayar ◽  
Karl Roberts ◽  
Emmanuel Olivier ◽  
Eric E. Bouhassira
Keyword(s):  
Red Blood Cells ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Blood Cells ◽  
Vascular System ◽  
Conflicts Of Interest ◽  
Direct Injection ◽  
Culture Method ◽  
Thrombocytopenic Purpura ◽  
Membrane Bound

Red Blood Cells (RBCs) have long been considered a potentially useful means of delivering drugs to the circulation because delivery through therapeutic RBCs as compared to direct injection in the plasma can lengthen the half-life of the therapeutic agent in the circulation, spatially restrict the drugs to the lumen of the cardio-vascular system, and shield the drug from the immune system. Despite some progress, loading the cells with therapeutically useful cargo remains technically challenging. We have recently developed PSC-RED, a chemically-defined scalable method to differentiate induced pluripotent stem cells (iPSCs) into unlimited numbers of enucleated cultured RBCs. This provides an ideal method to produce therapeutic RBCs since iPSCs can be genetically manipulated with powerful CRISPR-based technologies. ADAMTS13, whose deficiency is responsible for congenital and acquire Thrombotic Thrombocytopenic Purpura (TTP) is a good target as a therapeutic that could be delivered through drug-carrying RBCs because large amounts of plasma concentrate, or more recently recombinant proteins, are necessary to treat TTP. We report here we have produced engineered erythroid cells that contains globin-LCR driven ADAMTS13 fusion transgenes inserted at safe harbor AAVS1, and that these cells express a membrane bound version of an inhibitor-resistant version of ADAMTS13. We show using flow cytometry that the fusion protein is express at high levels, and using a FRET assay that detect cleavage of the von Willebrand cognate site, that the membrane bound ADAMTS13 is enzymatically active. Comparison of enzymatic activity with plasma concentrate suggests that about 50 billion engineered ADAMTS13-cRBCs would be sufficient to deliver an amount of ADAMTS13 equivalent to 2 liters of plasma concentrate. This suggests that a transfusion of about 10 mL of ADAMTS13-RBCs could be therapeutic for congenital and acquired TTP. The number of cRBCs necessary to treat even a few patients is very large. This has been considered a major obstacle to the development of treatment based on in vitro produced RBCs because of the volume of culture that is necessary to produce the cells. We also report that we have developed a culture method based on holo-fiber bioreactors that allows the production of cRBCs at a density of 5.108 cell/mL which is sufficient to produce enough cells to performed small clinical trials. Disclosures No relevant conflicts of interest to declare.

MODIFICATION OF THE TECHNIC OF TREATING ERYTHROBLASTOSIS FETALIS BY EXCHANGE TRANSFUSION

PEDIATRICS ◽  
1951 ◽  
Vol 8 (1) ◽  
pp. 117-127
Author(s):  
ALEXANDER S. WIENER ◽  
IRVING B. WEXLER
Keyword(s):  
Statistical Analysis ◽  
Mortality Rate ◽  
Red Blood Cells ◽  
Clinical Features ◽  
Blood Cells ◽  
Exchange Transfusion ◽  
Red Cells ◽  
Simplified Method ◽  
Adult Plasma

A simplified method of treating erythroblastosis by exchange transfusion is described in which the patient is bled and simultaneously transfused with compatible packed red cells. A table is presented which gives the final hematocrit and percentage of inagglutinable red blood cells in the patient's body at the termination of the exchange transfusion, in relation to the patient's initial hematocrit and the amount of blood exchanged. An exchange transfusion with 100 to 150 cc. of packed cells appears to be adequate regardless of the severity of the anemia. Statistical analysis of the result of 106 exchange transfusions shows a progressive drop in mortality rate from 23.7% in 38 cases treated with 500 cc. of whole citrated blood, to 14.6 cc. in 48 cases treated with 1,000 cc. of whole citrated blood, and to 10.0% in 20 cases treated with 120 cc. of packed cells. While the improvement may be accidental, there is no doubt that exchange transfusion with packed cells is a simpler procedure which avoids the introduction into the patient's body of large amounts of adult plasma, and potentially toxic doses of citrate and calcium. Two cases are described in detail which have unusual serologic and clinical features and illustrate the types of problems which may arise when treating erythroblastotic babies.

scholarly journals Preoperative embolization of the splenic artery in patients that underwent splenectomy for immune thrombocytopenic purpura

2007 ◽  
Vol 22 (6) ◽  
pp. 470-473 ◽  
Author(s):  
PlínioCarlos Baú ◽  
Sílvio Adriano Cavazolla ◽  
Hamilton Petry Souza ◽  
Bernardo Garicochea
Keyword(s):  
Blood Cell ◽  
Red Blood Cells ◽  
Red Blood Cell ◽  
Immune Thrombocytopenic Purpura ◽  
Blood Cells ◽  
Splenic Artery ◽  
Red Blood Cell Transfusion ◽  
Preoperative Embolization ◽  
Surgical Team ◽  
Thrombocytopenic Purpura

Transfusion of platelets, red blood cells, or both is usually necessary immediately after splenic artery ligature in patients with immune thrombocytopenic purpura who undergo splenectomy. PURPOSE: To investigate whether preoperative embolization of the splenic artery reduced the need for transfusion of platelets, red blood cells, or both. METHODS: Twenty- seven consecutive patients that underwent splenectomy for purpura between October 1999 and March 2006 performed by the same surgical team were enrolled. The first 17 patients did not undergo embolization and were compared with the next 10 patients, who composed the embolization group. RESULTS: The platelet count in the embolization group rose from a mean 7000 u/µl before to 75000 u/µl after the procedure. There was no need for platelet or red blood cell transfusion in the embolization group; in the group without preoperative embolization, 11 patients (p=0.001) required platelet transfusion and 8 (p=0.01), red blood cell transfusion. CONCLUSION: Embolization of the splenic artery before splenectomy is a safe method to avoid blood transfusions in patients with ITP.

Thrombotic Thrombocytopenic Purpura and Hemolytic Uremic Syndrome Are Distinct Pathologic Entities

2003 ◽  
Vol 127 (7) ◽  
pp. 834-839 ◽  
Author(s):  
Gregory A. Hosler ◽  
Ana M. Cusumano ◽  
Grover M. Hutchins
Keyword(s):  
Adrenal Gland ◽  
Hemolytic Uremic Syndrome ◽  
Thrombotic Thrombocytopenic Purpura ◽  
Clinical Features ◽  
Disease Process ◽  
Johns Hopkins Hospital ◽  
Thrombocytopenic Purpura ◽  
Pathologic Features ◽  
Distinct Disease ◽  
Uremic Syndrome

Abstract Context.—Thrombotic thrombocytopenic purpura (TTP) and the hemolytic uremic syndrome (HUS) share many clinical features and have been difficult to separate into distinct entities. Histologic examination of organs from autopsied patients suggested that TTP and HUS have dissimilar lesions of different severity and distribution. Objective.—To perform a retrospective observational review of autopsied patients with TTP or HUS to compare the nature and severity of the lesions found. Design.—To examine the pathologic features of these conditions, we reviewed all cases among 51 350 indexed autopsies at The Johns Hopkins Hospital (Baltimore, Md) diagnosed with TTP or HUS, and included those showing multiple arteriolar thrombi or their sequela. Results.—The 56 cases that met the inclusion criteria fell into 2 distinct groups, based on distribution and severity of arteriolar lesions. In 25 patients classified as having TTP, platelet-rich thrombi were present—in decreasing severity—in heart, pancreas, kidney, adrenal gland, and brain. In 31 patients with HUS, fibrin/red cell–rich thrombi were present, largely confined to the kidney and often severe, and only 6 cases showed pancreas involvement, 4 adrenal gland involvement, 2 brain involvement, and 1 heart involvement. Conclusion.—Despite similar clinical features and therapeutic approaches, TTP and HUS each have a characteristic constellation of histopathologic findings. This observation suggests that TTP and HUS are 2 distinct disease entities with different pathophysiologies, and that they do not represent a spectrum of the same disease process.

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