Successful Treatment of Kasabach-Merritt Syndrome with Prednisone and Epsilon-Aminocaproic Acid

The Kasabach-Merritt syndrome is characterized by thrombocytopenia and localized coagulopathy associated with a hemangioma. Most techniques applied to eradicate the tumor or accelerate its involution (surgery, radiation therapy, embolization) are invasive and require transfusion of large amounts of blood products. In some cases, medical treatment is the only alternative. Efficacy of steroids and antifibronolytic agents has already been described, but even this approach is associated with the administration of blood products. We report two cases of infants with Kasabach-Merritt syndrome associated with cardiac and hepatic hemangiomas. At admission, both had signs of cardiac failure. They were successfully treated with prednisone and epsilon-aminocaproic acid (EACA). Blood products were not required once the diagnosis was made. These observations have important implications for the management of patients with Kasabach-Merritt syndrome because they show that even in severe cases blood transfusions can be avoided by the use of prednisone and EACA.     

Effect of Glucose on Plasma Glucagon,Growth Hormone,and Insulin in Exchange Transfusion

Exchange transfusions were performed on term normal-for-dates, and term small-for-dates infants using blood preserved with acid citrate and glucose or acid citrate alone. The plasma concentrations of glucose, free fatty acids, glycerol, insulin, glucagon, and growth hormone were measured in the donor blood and in blood from the infant at different times during the transfusion. The total amounts of the metabolites and hormones infused and removed from the infant were calculated. The infusion of glucose in blood preserved with acid citrate and glucose caused a rise in plasma glucose, insulin, and growth hormone levels and a fall in plasma free fatty acid levels. The plasma glycerol levels did not change significantly during transfusions with blood preserved with acid citrate and glucose, or acid citrate alone. In both types of exchange transfusion there was a similar gradual fall in plasma glucagon concentration and net loss of free fatty acid, glycerol, and glucagon. A net loss of growth hormone occurred which was greater in transfusions performed with blood preserved with acid citrate and glucose than in transfusions performed with blood preserved with acid citrate. In transfusions performed with blood preserved with acid citrate and glucose there was a net accumulation of glucose by the infant and a net loss of insulin.     

Thrombocytopenia absent radius syndrome associated with renal insufficiency

Thrombocytopenia absent radius (TAR) syndrome, associated with small, echogenic kidneys and subsequent end-stage renal disease, was seen in a full-term newborn. In this infant, renal insufficiency may have increased the known risk of bleeding that is associated with TAR syndrome. Renal abnormalities and dysfunction are unusual in TAR syndrome. When they are present, however, treatment with frequent transfusions and early dialysis may be necessary to prevent serious and life-threatening hemorrhagic episodes.     

Microangiopathic hemolytic anemia and thrombocytopenia in a neonate associated with a large placental chorioangioma.

The case of an infant born in association with a large chorioangioma of the placenta is presented. The maternal complications of polyhydramnios, preeclampsia, and premature labor are those described in the "syndrome" of a chorioangioma. The immediate neonatal course was unusually complicated by severe microangiopathic anemia with persistent thrombocytopenia and hemolysis which required three exchange blood transfusions. The relationship between the neonatal complications and the placental tumor is discussed in terms of possible pathophysiologic mechanisms.     

Development of Kasabach-Merritt syndrome following needle aspiration of a hemangioma.

A case of Kasabach-Merritt syndrome that developed following needle aspiration in an infant with hemangioma is presented. Enlargement of the tumor leading to severe respiratory distress accompanied by hemorrhages occurred within six to eight hours after this simple intervention. The symptoms continued despite repeated thrombocyte transfusions and prednisolone therapy. Radiotherapy and local intraarterial corticosteroid therapy were attempted and led to some improvement. It took approximately three months for the platelet count and one year for the clinical state to revert to normal. This case illustrates that minor trauma may lead to life-threatening symptoms in a young infant with congenital hemangioma.     

Reducing donor exposure in preterm infants requiring multiple blood transfusions.

Preterm infants frequently require multiple blood transfusions. Traditionally, ''fresh'' (less than seven days old) blood has been used but this often results in transfusions from multiple donors. To reduce donor exposure the policy for top-up transfusions was changed. A unit of blood under five days old with additional satellite packs was ordered for each infant and used up to its expiry date, allowing up to eight transfusions from a single donation to be given. The mean (SD) number of transfusions per infant in 43 infants transfused according to previous policy and in 29 transfused according to the new policy was similar at 5.6 (4.0) and 5.3 (3.1), respectively. However, donor exposure fell following the change in policy from 4.9 (3.5) to only 2.0 (0.9). Only one infant was exposed to more than three donors compared with 24 infants in the control group. Plasma potassium concentrations were not significantly different following transfusion of blood stored for up to 33 days. This simple change in policy has reduced donor exposure in infants requiring multiple top-up transfusions.     

Tranexamic acid in the prevention of periventricular haemorrhage.

Increased fibrinolytic activity in the ganglionic eminence of the preterm human brain has been proposed as a factor in the aetiology of periventricular haemorrhage. The effect of tranexamic acid in preventing periventricular haemorrhage was evaluated in 100 infants in a double blind, randomised controlled trial. Haemorrhages developed in 22 infants who received tranexamic acid and in 20 of those who received placebo. A significant reduction in fibrin degradation products in treated infants was seen. Our study suggests that excessive fibrinolytic activity is not an important factor in the aetiology of periventricular haemorrhage and that treatment with tranexamic acid will not prevent its occurrence.     

Influence of Donor Blood Temperature on Metabolic and Hormonal Changes During Exchange Transfusion

Measurements of plasma glucose, free fatty acid, glycerol, insulin, growth hormone, and glucagon were made during exchange transfusions performed with blood heated to 36·5 to 38·5 °C or cooled to 10 to 13 °C. Term, normally grown infants suffering from rhesus incompatibility were studied. 10 received warm transfusions and 9 cold transfusions. Cold transfusions caused a progressive fall in rectal, umbilical vein, and skin temperature, whereas warm transfusions caused little change in body temperature. Infants receiving cold transfusions had a greater net positive balance of glucose and a smaller net negative balance of free fatty acids than those receiving warm transfusions. Other metabolic and hormonal responses to exchange transfusion were similar in the two groups.It was concluded that while exchange transfusion performed with cold blood did cause thermal stress to the infant, the metabolic consequences were slight and were mitigated by the glucose in the donor blood. There was no evidence that insulin, growth hormone, or glucagon played a part in the response of the newborn infant to cold.     

Gastric angiodysplasia in a child with Bernard-Soulier syndrome: efficacy of octreotide in long-term management

Gastrointestinal angiodysplasia in association with Bernard-Soulier syndrome has been previously described in adults. The authors report on a 14-year-old boy presenting with massive upper gastrointestinal bleeding due to a large gastric angiodysplasia, in whom medical history and laboratory investigations were consistent with Bernard-Soulier syndrome. The vascular lesion was so widespread that surgical or endoscopic therapy was not considered. Therefore, treatment with octreotide, a somatostatin analog, was commenced, following a short course of tranexamic acid and proton pump inhibitor. During the 16-month follow-up with octreotide therapy, no occult or gross bleeding occurred. This case illustrates the utility of using octreotide for the long-term treatment of children with bleeding disorders and angiodysplasia.     

D-Lactic acidosis: a treatable encephalopathy in pediatric patients

A 20-month old infant, who had short bowel syndrome following extensive surgery for a mid gut volvulus, developed hyperchloremic acidosis, with a large anion gap after enteral feeding was instituted. The organic acidosis was at least partly due to an increased concentration of D-lactic acid. This patient, as did five other pediatric patients, presented with a typical encephalopathy syndrome. Early recognition of this syndrome and treatment with an intestinal antibiotic and discontinuation of enteral feeding enabled prompt correction of the hyperchloremic acidosis and a rapid clinical recovery.     

Graft versus host reaction in an infant with DiGeorge syndrome

An infant with complete DiGeorge syndrome was treated with blood transfusions and fresh frozen plasma because of severe septicemia and anemia. 9 weeks after the first transfusion and 2 weeks after administration of fresh frozen plasma he died of acute graft-versus-host disease. The blood products were routinely irradiated with 25 gray, the fresh frozen plasma was not irradiated. The diagnosis of GvHD was confirmed on autopsy. All cellular blood products including fresh frozen plasma which are used in the treatment of immuno-deficient patients have to be irradiated to avoid GvHD. Currently 50 gray are recommended.     

Spindle cell (Kaposiform) hemangioendothelioma with Kasabach-Merritt syndrome in an infant: Successful treatment with α-2A interferon

A two-month-old infant developed a vascular tumor of the right flank which, upon biopsy, proved to be a spindle cell hemangioendothelioma. The increased capillary bed characterizing the neoplasm caused a severe thrombocytopenia together with a consumption coagulopathy (Kasabach-Merritt syndrome). The patient, who was dependent on platelet transfusions, improved quickly after interferon α-2a was given at the dosage of 3,000,000 U/m², with resolution of the Kasabach-Merritt syndrome after three weeks and a 75% decrease of the tumor volume within three months of treatment. Med. Pediatr. Oncol. 28:358–361, 1997. © 1997 Wiley-Liss, Inc.     

Avoidance of red blood cell transfusion in an extremely preterm infant given recombinant human erythropoietin therapy

To avoid red blood cell (RBC) transfusions, recombinant human erythropoietin (rHuEPO) was given to an infant born at a gestation of 26 weeks and a birthweight of 830 g to parents who were Jehovah's Witnesses. The infant had hyaline membrane disease and required 52 days of assisted ventilation and 19 days of oxygen therapy. He received theophylline therapy for 61 days for recurrent apnoea and bradycardia. He developed bilateral intraventricular haemorrhage (IVH) and left-sided periventricular leucomalacia (PVL). Intravenous rHuEPO was started on day 1 at 200 U/kg per day for 1 month followed by subcutaneous rHuEPO 400 U/kg three times a week for 6 more weeks, supplemented with Vitamin E, folic acid and iron. Blood sampling was kept to a minimum and non-invasive blood-gas monitoring was used consistently. Consequently, the estimated cumulative volume of blood loss from sampling was only 21 mL during his hospital stay. His haemoglobin (Hb) was 150 g / L at birth and this fell to below 100 g / L from day 25 onwards. His lowest leucocyte count was 3.6x10⁹/L. He was discharged on day 83 with a Hb of 95 g/L, Hct of 29%, reticulocyte count of 2.8% and weight of 2400 g. At a postnatal age of 3 months, he had a Hb of 113 g/L. At 6 months, investigations showed: Hb 121 g/L, haematocrit 33%, reticulocyte 1% and a weight of 4.4 kg. He was readmitted to hospital once for an episode of vomiting and follow up to date showed developmental delay. Concerns remained whether the withholding of RBC transfusion in the infant was related to his IVH, PVL, prolonged recurrent apnoea and bradycardia and poor weight gain. Although rHuEPO therapy shows promise in reducing the need for RBC transfusions, its overall efficacy and safety remain to be proven and its routine use in preterm infants has to be weighed up against the potential benefits and risks of RBC transfusions.     

Successful management with interferon alpha-2a after prednisone therapy failure in an infant with a giant cavernous hemangioma

A giant cavernous hemangioma of the left arm with severe thrombocytopenia and consumptive coagulopathy was observed in a neonate. Initial treatment with prednisone, platelet transfusions, and clotting replacement failed to control the bleedings. The child was then treated with daily subcutaneous infusions of interferon alpha-2a. Coagulopathy rapidly improved and transfusions were drastically reduced. The hemangioma regressed progressively and disappeared after 4 months of treatment. Med. Pediatr. Oncol. 28:213–215 © 1997 Wiley-Liss, Inc.     

Cyclosporine in the treatment of red cell aplasia

A 4-year-old girl was diagnosed to have pure red cell aplasia. For 5 years, she received multiple packed red cell transfusions (average 11/year). Several therapeutic maneuvers were attempted including prednisone, oxymethalone, plasmaphoresis, high dose methyl prednisolone-to no avail. Finally, cyclosporine was administered for 5 months. This increased her reticulocyte count and cured the disease. For the last 2 years she has been in excellent health and has not required any further transfusions. The possible mechanism of action of cyclosporine in this disease is discussed.     

METABOLIC CONSEQUENCES IN VERY LOW BIRTH WEIGHT INFANTS TRANSFUSED WITH OLDER AS-1-PRESERVED ERYTHROCYTES

Our study evaluated the biochemical and physiologic changes in extremely premature infants receiving AS - 1 - preserved packed red blood cells (PRBC) stored 5 to 21 days, including increase in hemoglobin, maintenance of euglycemia, and normal serum potassium levels, acid - base balance, and urine output. Twelve infants (birth weight 775 +/- 127 g) with gestational age of 25 +/- 1 weeks received small volume replacement transfusions with 17 mL /kg PRBC preserved in an AS- 1 dedicated unit. Infants were monitored throughout the transfusions and blood samples were obtained pre - and postransfusion for comparison by Student's t - test. The infants remained clinically stable throughout the transfusions and had an increase in hemoglobin of 2.5 g /dL. There was no change in urine output. Serum ammonia lactate dehydrogenase (LDH), and glucose levels were altered as predicted. There were also small, but clinically insignificant, changes in serum potassium, bicarbonate, total bilirubin, and creatinine. Euglycemia was maintained in 84 % of the transfusions. We concluded that small volume transfusions with PRBC stored up to 21 days in AS - 1 can be used in the very low birth weight infant without apparent detriment when extensive metabolic criteria are examined.     

The Role of Platelets and ε‐Aminocaproic Acid in Arthrogryposis,Renal Dysfunction,and Cholestasis (ARC) Syndrome Associated Hemorrhage

Arthrogryposis, renal dysfunction, and cholestasis (ARC) syndrome is a rare disorder associated with platelet abnormalities resembling gray platelet syndrome. Affected patients have normal platelet numbers but abnormal morphology and function. Bleeding symptomatology ranges from postprocedural to spontaneous life‐threatening hemorrhage. We report a patient with ARC syndrome and compound heterozygous mutations in VPS33B (vacuolar protein sorting 33B) who presented with significant bleeding requiring numerous admissions and transfusions. She was treated with prophylactic platelet transfusions and ε‐aminocaproic acid. This was well‐tolerated and significantly decreased transfusion requirements and admissions for bleeding. Our experience provides support for consideration of prophylactic measures in these patients as well as the possibility of using prophylaxis in related disorders.     

Neonatal paracetamol poisoning: treatment by exchange transfusion.

The metabolism and excretion of paracetamol was studied in an infant of 29 weeks'' gestation who was exposed to the drug when his mother ingested 32.5 g 16 hours before delivery. We have confirmed that sulphation is the major pathway and that the mixed function oxidase system is sufficiently active at this gestational age to produce hepatotoxic metabolic products. As most of the recognised drug treatments for paracetamol poisoning seemed unsuitable in this case, the infant was treated with exchange transfusions.     

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??Objective To observe the effect of mizoribine??MZR?? in the treatment of children with frequently relapsing primary nephrotic syndrome. Methods Totally 21 children with frequently relapsing nephrotic syndrome were treated with mizoribine?? and another 24 children were included as the control group. Observe the changes of relapse number??dosage of prednisone?? glomerular filtration rate?? serum albumin?? urine protein?? uric acid and other indicators. Results After the treatment with mizoribine??the recurrence rate and the effective dosage of prednisone decreased?? serum albumin level rose?? and primary urinary protein decreased?? but there were no significant changes in glomerular filtration rate and uric acid level. Conclusion The mizoribine is effective in treatment of children with frequently relapsing primery nephrotic syndrome .     

Pseudo-Zellweger syndrome: deficiencies in several peroxisomal oxidative activities

We describe an infant girl with a clinical, chemical, and pathologic syndrome remarkably similar to Zellweger cerebrohepatorenal syndrome but whose liver parenchymal cells contained abundant peroxisomes. Peroxisomal L-alpha hydroxy acid oxidase, catalase, and the plasmalogen synthesizing enzyme dihydroxy acetone phosphate-acyl transferase activities were normal; other peroxisomal enzymatic activities, including fatty acyl-CoA oxidase and D-amino acid oxidase, were reduced by 80% to 85%. Oxidation of bile acids and pipecolic acid was also deficient. Autopsy revealed the presence of neuronal heterotopia, renal cortical cysts, adrenal atrophy, and accumulation of very long chain fatty acids. The clinical and pathologic features of this case of "pseudo-Zellweger syndrome" reflect a deficiency in multiple peroxisomal activities rather than a defect in peroxisomal biogenesis. The deficient enzymatic activities require flavin adenine dinucleotide, and the underlying defect may be in the utilization of this cofactor.