Baris Kuskonmaz

Secondary hemophagocytic lymphohistiocytosis in Turkish children.

Between January 1998 and January 2005, a total of 18 children 2 weeks–72 months of age were diagnosed as having secondary hemophagocytic lymphohistiocytosis. The frequency of secondary hemophagocytic lymphohistiocytosis among total hospitalized patients during this period was 0.05% (18 of 34,250). Of the 18 patients, 8 (44.5%) had bacterial infections; cytomegalovirus and Epstein-Barr virus infections were present in 5 (28%) and 1 (5.5%), patient, respectively. Leishmaniasis was diagnosed in 2 patients (11%), and herpes simplex virus was diagnosed in 2 patients (11%). Six patients died during treatment, and 1 patient was lost to follow-up. The survival rate was 61%.

Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching

Haematopoietic stem cell transplantation (HSCT) remains the best therapeutic option for many acquired and inherited paediatric haematological disorders. Unfortunately, the probability of finding an HLA matched donor is limited. An alternative technique is PGD combined with HLA matching, which offers the possibility of selecting unaffected embryos that are HLA compatible with the sick child, with the aim of possible use of stem cells from the resulting baby in future. Since the first successful report for Fanconi anaemia a decade ago, the therapeutic success of this technique was reported in a few cases and for a limited number of disorders. Here, we report full recovery of 44 sick children who received HSCT from healthy infants conceived after pre-implantation HLA matching for the following 10 indications; beta-thalassaemia, Wiskott-Aldrich syndrome, Fanconi anaemia, sickle cell anaemia, acute myeloid leukaemia, acute lymphoblastic leukaemia, Glanzmanns thrombasthaenia, Diamond-Blackfan anaemia, X-linked adrenoleukodystrophy and mucopolysaccharidosis type I. No serious complications were observed among recipients and donors. Graft failure occurred in four children with beta-thalassaemia where a second HSCT was planned. Preimplantation HLA matching is a reliable technique and provides a realistic option for couples seeking treatment for an affected child when no HLA-matched donor is available.

Relationship of lower breastfeeding score and problems in infancy.

BACKGROUND AND METHODS We undertook a descriptive study in 2-month-old healthy infants to determine the factors that affect breastfeeding score. Mothers breastfeeding was evaluated and scored according to the World Health Organization/UNICEF B-R-E-A-S-T Feeding Observation Form. RESULTS The breastfeeding score (BFS) was higher in female than male babies (p=0.005). The babies with regurgitation had lower BFS than the babies without (p=0.016). The BFS was lower in babies who had repeated, without cause, inconsolable crying than in those without such crying (p<0.004). When the crying was problematic for the family, BFS was lower (p=0.028). Babies who had another sibling with a history of colic had a lower BFS (p=0.038). A low BFS was associated with short duration of night sleeping (p=0.032). CONCLUSIONS A decreased BFS may be a risk factor or indicator for infant crying, regurgitation, and short sleeping duration. As a result, tracking the BFS and appropriate breastfeeding intervention during the newborn period may assist in decreasing the frequency of regurgitation and infant crying.

The effect of glutamine supplementation on hematopoietic stem cell transplant outcome in children: A case-control study

Abstract:  HSCT associated morbidity and mortality is usually attributed to high‐dose chemotherapy/radiotherapy regimens used for conditioning. Glutamine (Gln), a conditionally essential amino acid during severe catabolic states, has been shown to have favorable effects in patients with malignancies and in those undergoing HSCT. However, controversy exists regarding its routine use. Studies in children investigating gln supplementation are very limited. In the present study, including 21 gln‐supplemented and 20 control pediatric patients, gln supplementation was shown to reduce the duration of fever and decrease the incidence of SOS during the HSCT course. In addition, a decrease in drug‐related toxicity and a trend toward reduced incidence of severe mucositis were observed.

Hepatitis B immunoglobulin in combination with lamivudine for prevention of hepatitis B virus reactivation in children undergoing bone marrow transplantation

Abstract:  There is little information in literature about the use of hepatitis B immunoglobulin (HBIg) in recipients of bone marrow transplantation (BMT). Here, we report two children who received IV HBIg (Hepatect‐CP) and lamivudine treatment during BMT course for either patient or donor hepatitis B virus (HBV) viremia. A four‐year‐old girl underwent a fully human leukocyte antigen‐matched allogeneic BMT for thalassemia major from her mother positive for hepatitis B surface antigen (HBsAg). A 12‐yr‐old boy with chronic myeloid leukemia, positive for HBsAg and HBV‐DNA received a fully HLA‐matched allogeneic BMT from his sister in the first chronic phase of the disease. HBIg was successfully used in both cases to prevent HBV reactivation of the recipients. The results of our observations are encouraging and we suggest that HBIg in combination with lamivudine may be used in such cases especially in post‐transplant early period to prevent HBV reactivation.

Successful treatment of severe myasthenia gravis developed after allogeneic hematopoietic stem cell transplantation with plasma exchange and rituximab

Myasthenia gravis is among the rare complications after allogeneic hematopoietic stem cell transplantation and is usually associated with chronic GVHD. Herein, we report a 2‐year and 10 months of age female with Griscelli syndrome, who developed severe myasthenia gravis at post‐transplant +22nd month and required respiratory support with mechanical ventilation. She was unresponsive to cyclosporine A, methylprednisolone, intravenous immunoglobulin, and mycophenolate mofetil and the symptoms could only be controlled after plasma exchange and subsequent use of rituximab, in addition to cyclosporine A and mycophenolate mofetil maintenance. She is currently asymptomatic on the 6th month of follow‐up. Pediatr Blood Cancer 2014;61:928–930.

DEFERASIROX USE AFTER HEMATOPOIETIC STEM CELL TRANSPLANTATION IN PEDIATRIC PATIENTS WITH β-THALASSEMIA MAJOR: Preliminary Results

There are limited data on the posttransplantation pharmacological treatment of iron overload in ex-thalassemic patients and the current approach is phlebotomy. The authors chelated 2 ex-thalassemic patients after hematopoietic stem cell transplantation with deferasirox for 6 and 24 months. Although serum ferritin levels decreased, cardiac and hepatic iron load, measured by T2* magnetic resonance imaging (MRI), showed decrease in iron overload in these organs. The drug was tolerated well by both patients and no adverse effect on donor hematopoiesis was observed. This preliminary study demonstrates that deferasirox is well tolerated in these patients and will be a good potential therapy when more data have been obtained from larger studies.

Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II

CDA are a group of inherited, rare diseases that are characterized by dyserythropoiesis and ineffective erythropoiesis associated with transfusion dependency in approximately 10% of cases. For these latter patients, the only curative treatment is HSCT. There are very limited data on HSCT experience in this rare disease. Herein, we report a five‐yr six‐month‐old girl with compound heterozygous mutations in SEC23B gene, who was diagnosed to have CDA type II and underwent successful HSCT from her matched sibling donor.

Number of erythrocyte transfusions is more predictive than serum ferritin in estimation of cardiac iron loading in pediatric patients with acute lymphoblastic leukemia.

BACKGROUND Transfusions with packed erythrocytes is a common practice in pediatric patients with acute lymphoblastic leukemia (ALL) who are on chemotherapy. Since there is no physiological excretion mechanism for iron, the iron related to erythrocyte transfusions accumulates and may contribute to late cardiac, hepatic and endocrine complications in these patients. PROCEDURE In order to evaluate the iron burden among pediatric patients with ALL and define the risk factors associated with higher iron loading, we evaluated 79 pediatric patients with ALL (36 were off-therapy). Cardiac and hepatic T2* were ordered to a total of 22 (28%) patients who were either transfused with erythrocytes ≥ 10 times (n=11; 50%), had serum ferritin (SF) ≥ 1000 ng/ml (n=2; 9.1%) or both (n=9; 40.9%). RESULTS Half of the patients who were screened by T2* MRI had hepatic T2*<7 ms and six (27%) of the patients had cardiac T2*<20 ms, indicating iron loading. Patients who had serum ferritin <1000 vs ≥ 1000 ng/ml had median cardiac T2* values of 28.3 ms (15-40) vs 21 (7.9-36), (p=0.324); whereas hepatic T2* of 10.8 (5.32-27) vs 4.7 (2.2-36), (p=0.017). Patients who had erythrocyte transfusion <10 vs ≥ 10 times had median cardiac T2* values of 34 ms (28-38) vs 23 (7.93-40), (p=0.021); whereas hepatic T2* of 13.6 (6.6-36) vs 5.32 (2.2-27), (p=0.046). CONCLUSIONS Our results indicate that pediatric patients with ALL should be screened for transfusional iron load and the amount of erythrocyte transfusions seems to be a more reliable indication than serum ferritin levels to detect cardiac iron loading in these patients.

TRANSFUSION-TRANSMITTED VIRUS PREVALANCE IN TURKISH PATIENTS WITH THALASSEMIA

In hematology patients on chronic transfusion regimes, liver diseases are frequent, and mostly related to the agents transmitted by blood products and concominant iron deposition in liver. Besides hepatitis B (HBV) and C (HCV) viruses, new viral agents like hepatitis G virus (HGV) and TorqueTeno virus (TTV) are identified in these patients, although their association with any pathology or disease is not yet proved. In the present work, the authors studied the clinical importance of TTV in Turkish multitransfused patients with thalassemia. Forty-six healthy and 57 thalassemic patients were enrolled in the study. TTV was detected in serum samples by 3′-UTR nested PCR. Transaminase and ferritin levels, hepatitis B and C virus markers and number of transfusions were interpreted for possible association with TTV infection. As a result, TTV was detected in 63% of the thalassemia and 54% of the control patients. Prevalence of TTV infection, clinical features, laboratory data, and annual transfusion numbers of TTV-positive and -negative patients were not observed to be statistically significant. In conclusion, in Turkish patients with thalassemia, TTV infection cannot be considered as a risk factor for liver disease.