Kleebsabai Sanpakit

Mahidol University, Krung Thep, Bangkok, Thailand

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Publications (24)34.87 Total impact

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    ABSTRACT: In this study we report eight compound heterozygotes for mutations in the key erythroid transcription factor Krüppel-like factor 1, (KLF1) who presented with severe, transfusion-dependent hemolytic anemia. In most cases the red cells were hypochromic and microcytic consistent with abnormalities in hemoglobin synthesis. In addition, in many cases, the red cells resembled those seen in patients with membrane defects or enzymopathies; known as chronic non-spherocytic hemolytic anemia (CNSHA). Analysis of RNA and protein in primary erythroid cells from these individuals provided evidence of abnormal globin synthesis, with persistent expression of fetal hemoglobin and, most remarkably, expression of large quantities of embryonic globins in post-natal life. The red cell membranes were abnormal, most notably expressing reduced amounts of CD44 and consequently manifesting the rare In(Lu) blood group. Finally, all tested patients showed abnormally low levels of the red cell enzyme pyruvate kinase; a known cause of CNSHA. These patients define a new type of severe, transfusion dependent CNSHA caused by mutations in a trans-acting factor (KLF1) and reveal an important pathway regulating embryonic globin gene expression in adult humans.
    Blood 01/2014; · 9.78 Impact Factor
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    ABSTRACT: Improving outcomes among class 3 thalassemia patients receiving allogeneic hematopoietic stem cell transplant (HSCT) remains a challenge. Prior to HSCT, patients who were > 7 years old and had a live size > 5 cms constitute what the Center for International Blood and Marrow Transplant Research (CIBMTR) has defined as a very high-risk subset of a conventional high risk class 3 group (here referred to as Class 3 HR). We performed HSCT in 98 patients with related and unrelated donor stem cells. Seventy-six of the patients with age < 10 years received the more conventional myeloablative conditioning (MAC) regimen (cyclophosphamide, busulfan , + fludarabine), the remaining twenty two patients with age > 10 years and hepatomegaly (“Class 3 HR”), and in several instances additional comorbidity problems, underwent HSCT with a novel reduced toxicity conditioning (RTC) regimen (fludarabine and busulfan).We then compared the outcomes between these two groups(MAC vs RTC); Event-free survival(86% vs 90%), and overall survival (95% vs 90%) were not significantly different between the respective groups, however, there was a higher incidence of serious treatment-related complications in the MAC-group and while we experienced 6 graft failures in the MAC group (8%), there was none in the RTC group. Based on these results, we suggest, 1) that high risk class 3 thalassemia patients can safely receive HSCT with our novel RTC regimen and achieve the same excellent outcome as low/standard-risk thalassemia patients who received the standard MAC regimen, and further, 2) that this novel RTC approach should be tested into the low/standard-risk patient population.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 01/2014;
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    ABSTRACT: Medical records of 30 children with renal tumor diagnosed at Siriraj Hospital during 1996 to 2007 were reviewed. Mean age at diagnosis was 36 months; male to female ratio was 1.7:1. Clinical manifestations included abdominal mass (96.7%), hypertension (40.0%), abdominal pain (36.7%), hematuria (26.7%), postrenal obstruction (16.7%), and proteinuria (13.3%). Eight patients had Denys-Drash malformations, WAGR, Dandy-Walker malformation, or genitourinary anomalies. Twenty-seven patients (90%) had Wilms tumor. Sixteen patients were stage 3 or more at diagnosis. Rhabdoid tumor was found in 3 patients. All patients received chemotherapy, 13 patients also received radiation therapy. Acute complications included febrile neutropenia (44.4%), hypokalemia (37.0%), hyponatremia (29.6%), Fanconi syndrome (11.1%), urinary tract Infection (10.0%), and acute renal failure (7.4%). Mean follow-up time was 57.2 months. Ten patients died from progressive disease. Five-year patient survival was 69.7%. Two patients had chronic kidney disease. One of these had Denys-Drash malformations. Both patients received ifosfamide-carboplatin-etoposide protocol and abdominal radiation. Antihypertensive medications were needed in 9 patients for a mean duration of 164 days. None had persistent proteinuria or hematuria. No difference was found among mean estimated glomerular filtration rate at diagnosis, 1 year after treatment, and at last follow-up. Long-term follow-up, especially renal function, is recommended.
    Journal of Pediatric Hematology/Oncology 11/2013; 35(8):610-3. · 0.97 Impact Factor
  • Kleebsabai Sanpakit, Vip Viprakasit
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    ABSTRACT: Transfusion-dependent Hb H disease is rarely reported. In the majority of patients, it is caused mainly by α-thalassemia from deletions of 2 linked α-globin genes and nondeletional mutations. Previously, we had described 2 unrelated Thai patients with this condition because of compound heterozygosity of SEA-type deletion (--/) and a novel nucleotide mutation: a thymine insertion at codon 131 of the α1 gene, namely, Hb Pak Num Po (Hb PNP, αα). We herein describe the identification of 4 additional patients with Hb PNP with a broader genotype/phenotype spectrum and provide an overview of clinical management approaches including stem-cell transplantation.
    Journal of Pediatric Hematology/Oncology 10/2013; · 0.97 Impact Factor
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    ABSTRACT: We retrospectively analysed the outcomes of 127 children with acquired severe aplastic anaemia (SAA) who had received haematopoietic stem cell transplantation (HSCT) between 2000 and 2011 in one of the 10 Asia Pacific institutions. Fifty-three were matched sibling donor (MSD) and 74 were alternative donor (AD), including 22 matched unrelated donor, 32 mismatched unrelated donor and 20 mismatched related donor. With a median follow up 45·5 months (13-139) and when compared to the MSD group, AD recipients had more grade II-IV acute graft-versus-host disease (aGVHD; 14·3% vs. 32·8%, P = 0·029), but similar grade III-IV aGVHD (10·2% vs. 12·5%, P = 0·774), graft failure (GF) (15·1% vs. 15·5%, P = 0·658) and 5-year overall survival (90·6% vs. 83·7%, P = 0·251). As a source of stem cell, peripheral blood stem cells (PBSC) resulted in less GF (18% vs. 9·1% P = 0·013), similar grade II-IV aGVHD (28·1% vs. 17·4%, P = 0·258), chronic GVHD (25·8% vs. 29·3%, P = 0·822) and similar outcomes (89·7% vs. 82·4%, P =0 ·665) when compared to bone marrow (BM). In univariate analysis, GF (P < 0·001) and grade II-IV aGVHD (P = 0·009) were predictors of poor survival. In multivariate analysis, only GF was associated with poor survival (P = 0·012). The outcome of AD and PBSC HSCT were comparable to that of MSD and BM HSCT in the Asia Pacific region.
    British Journal of Haematology 06/2013; · 4.94 Impact Factor
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    Pediatrics International 06/2012; 54(3):e19-21. · 0.88 Impact Factor
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    ABSTRACT: Introduced in 1988 by Kaneko and colleagues, selective ophthalmic arterial infusion of chemotherapeutic drug has recently gained more interest among retinoblastoma experts worldwide. The report showed that the procedure could be repeated up to 12 treatments without serious side effects. We report a 4-year-old girl with bilateral retinoblastoma. The left eye was enucleated for the group E disease. The right eye started with 3 retinal tumors (group C) was treated with systemic chemotherapy plus local therapy. Seven months after the last cycle of chemotherapy, the tumor recurred close to the fovea. Systemic chemotherapy was reinitiated without success. To avoid aggressive cryotherapy and external-beam radiotherapy, selective ophthalmic arterial infusion of chemotherapeutic drugs was performed for 15 sessions. The tumor responded partially without evidence of drug-induced retinal toxicity by the electroretinogram. Minor irregularities of the inner wall of supraclinoid portion of the internal carotid artery were observed only at the sixth session. Narrowing of the vascular lumen occurred on the last 2 sessions. We demonstrate that this technique when performed repeatedly could result in the anatomic changes of the small blood vessel. Careful follow-up is necessary for early detection of any serious consequences.
    Journal of Pediatric Hematology/Oncology 05/2012; 34(6):e218-21. · 0.97 Impact Factor
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    ABSTRACT: MRI-R2* has been accepted as a clinical tool for monitoring iron overload in thalassemia patients, especially for monitoring liver iron concentration (LIC). The most optimal and practical method of analysis however, is still open to further investigations. Our objective was to investigate intra- and intersite observer variability of the pixel-wise method for liver R2* analysis in thalassemia patients using a mono-exponential with a constant offset model. We performed 88 liver R2* measurements on 72 thalassemia major patients. A single breath-hold multi-echo gradient-echo sequence was acquired and analyzed at both the reference (REF) and local (LOC) sites. The analysis defined the region of interest in the whole liver parenchyma, excluding the great vessels, and were reported as median values. The R2* values from the REF and LOC were statistically comparable for all comparisons. The intrasite and intersite observer variation were 0.75% (less than 0.9%) and 2.5%, respectively, both of which are comparable to previous reports, but substantially lower than conventional region-based approaches. The low variation of the R2* also yielded excellent variation in the tabulated hepatic iron content. However, caution is required when comparing the results to different implementation methods and appropriate evaluation and validation of methodology for any new scan site is essential before its clinical use.
    Hematology/ Oncology and Stem Cell Therapy 01/2012; 5(2):91-5.
  • Pediatrics International 10/2011; 53(5):784-7. · 0.88 Impact Factor
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    ABSTRACT: Assess the use of N terminal pro brain natriuretic peptide (NT-pro BNP) to early diagnose ventricular dysfunction in doxorubicin-administered children. Fifty-five cancer patients who received accumulative dose of doxorubicin <300 mg/m2 (group 1), 49 cases with accumulative dose > or = 300 mg/m2 (group 2) and 52 cases as a control group (group 3) were included in the study. Electrocardiogram, chest roentgenogram, echocardiogram, and serum NT-pro BNP were studied. At age 1-10 years, there were significantly higher NT-pro BNP in group 2 than group 1 (384 +/- 291 vs. 92.2 +/- 89 pg/ml; p = 0.001), and than group 3 (79 +/- 92 pg/ml; p = 0.001). Patients with NT-pro BNP level > 1 SD of the control group were more likely to have abnormal > or = 2 echocardiographic parameters of left ventricular diastolic dysfunction than patients with NT-pro BNP < or = 1 SD (OR = 3.8, 95% CI 1.18-12.5). Patients in group 2 were more likely to have abnormal > or = 2 parameters of left ventricular diastolic dysfunction than patients in group 1 (OR = 2.8, 95% CI 1.07-7.7) and more likely to have NT-pro BNP >1 SD than group 1 (OR = 8, 95% CI 1.96-38.4). There were association of NT-pro BNP > 1 SD, accumulative dose of doxorubicin > or = 300 mg/m2, and early left ventricular diastolic dysfunction by echocardiogram. Serum NT-pro BNP > 1 SD has a high probability to diagnose early doxorubicin-induced cardiomyopathy in patient 1-10 years old.
    Journal of the Medical Association of Thailand = Chotmaihet thangphaet 11/2009; 92(11):1450-7.
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    ABSTRACT: Ectopic ACTH syndrome is a very rare cause of pediatric Cushing's syndrome. And if present, bronchial or thymic carcinoids predominate as causes. We hereby demonstrate a first case report of ACTH-producing ovarian steroid cell tumor, NOS, causing ectopic ACTH syndrome in a prepubertal girl.
    Endocrine 03/2009; 35(2):132-5. · 2.25 Impact Factor
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    ABSTRACT: Since 1997, our institute has used neoadjuvant chemotherapy for intraocular retinoblastoma. However, some of the patients showed signs of recurrence months to years later. We therefore attempted to determine the prognostic factors of treatment outcomes and survival in our patients. We reviewed 90 patients treated from 1997 to 2006. The following information was recorded: demographic and ophthalmological data, clinical classification, histopathological data, and treatment methods and outcomes. Enucleation was avoided in two of 57 eyes in the unilateral group. Sixteen of 54 eyes in the bilateral group were salvaged by systemic chemotherapy with local treatment. There was no difference in histopathological findings between the two groups. Nine of 57 patients in the unilateral group demonstrated poor outcomes, compared with four of 27 in the bilateral group. Significant poor prognostic factors for survival were optic nerve head invasion, orbital involvement, abnormal results on bone marrow aspiration, lumbar puncture, bone scan, and group E or F classification. The 15% mortality rate in our patients is slightly higher than that reported in developed countries. Delayed diagnosis and treatment is the main cause of avoidable deaths. Improving education of both clinicians and parents would increase survival rates in this potentially fatal disease.
    Japanese Journal of Ophthalmology 01/2009; 53(1):35-9. · 1.27 Impact Factor
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    ABSTRACT: Advances in hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major make the long-term outcome of these patients very important. Few data on long-term renal function of thalassemia patients are available. We evaluated the renal function in children after successful allogeneic HSCT for beta-thalassemia. Twenty-nine patients were included; the mean age at HSCT was 4.9 years. Mean follow-up time was 7.6 years. After HSCT, two patients developed acute renal failure and two had graft versus host disease. At last follow up, height standard deviation score (SDS) remained the same, but weight SDS had improved. Mean hemoglobin was 12.5 g/dl, and serum ferritin level was 545 ng/ml. All children had normal estimated glomerular filtration rate (GFR). One patient had hypertension and proteinuria, 10 years after HSCT. When comparing 39 children of the same age with beta-thalassemia of similar disease severity but who had not experienced HSCT, we found that the parameters of renal tubule function were better in patients that had undergone HSCT, as demonstrated by urine protein level (0.36 mg/mg creatinine vs 3.03 mg/mg creatinine, P < 0.001), osmolality (712 mosmol/kg vs 573 mosmol/kg, P = 0.006), N-acetyl-beta-D: -glucosaminidase (17.7 U/g creatinine vs 42.9 U/g creatinine, P = 0.045), and beta 2 microglobulin (0.09 microg/mg creatinine vs 0.13 microg/mg creatinine, P = 0.029). This study showed a low incidence of long-term renal impairment after HSCT and indicated that renal tubule function may be better in beta-thalassemia patients after HSCT.
    Pediatric Nephrology 08/2008; 24(1):183-7. · 2.94 Impact Factor
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    ABSTRACT: Severe combined immunodeficiencies (SCID) are disorders with impairment of humoral and cellular immune functions. The prognosis of disseminated bacillus Calmette-Guérin (BCG) infection in immunocompromised host is unfavorable since response to standard therapy is poor. We report a successful treatment of disseminated BCG infection with granulocyte colony stimulating factor (G-CSF) in a patient with severe combined immunodeficiency. The patient failed to response to intensive anti-tuberculous (anti-TB) therapy. After 2 months of G-CSF, in addition to anti-TB treatment, the clinical signs of disseminated BCG infection were improved. Since serious BCG infections in SCID are not uncommon in developing countries, where BCG vaccination is mandatory to all newborns, the combination of G-CSF and anti-TB drugs should be considered in immunocompromised patients with protracted mycobacterial infection.
    Asian Pacific journal of allergy and immunology / launched by the Allergy and Immunology Society of Thailand 04/2008; 26(1):71-5. · 0.79 Impact Factor
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    ABSTRACT: Juvenile xanthogranuloma, a histiocyte disorder, usually presents with a solitary cutaneous lesion. Juvenile xanthogranuloma with extracutaneous involvement is a rare disease in which significant morbidity and occasional deaths may occur. Monozygotic twins with congenital systemic juvenile xanthogranuloma who presented with multiple skin lesions, hepatosplenomegaly, liver failure, and bone marrow involvement were reported. The diagnosis of systemic juvenile xanthogranuloma was confirmed by histology and immunohistochemical stains of the skin with liver biopsies revealing dense infiltration of lymphohistiocytes with typical Touton giant cells staining positive for CD68 and negative for CD1a and S-100 protein. Both of them received systemic prednisolone 1 mg/kg/day which was gradually tapered off with time according to clinical and investigative responses. At the 17-month follow-up period, both patients showed remarkable regression in all symptoms and laboratory studies.
    Pediatric Dermatology 01/2008; 25(4):470-3. · 1.04 Impact Factor
  • American Journal of Kidney Diseases 11/2006; 48(4):668-73. · 5.29 Impact Factor
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    ABSTRACT: Dengue hemorrhagic fever (DHF) causing by dengue viral infection is endemic in Thailand and Southeast Asian countries where thalassemias are prevalent. Thalassemic patients are also at risk to acquire dengue viral infections and to develop DHF. However, they can have different clinical manifestations and complications as well as more severity than general population requiring special awareness for proper diagnosis and management. We reported 20 thalassemic patients (10 boys and 10 girls) with DHF admitted to Department of Pediatrics, Siriraj Hospital during 1977 to 2001. Their ages ranged from 2-16 years (average 9.5 years). These cases included 5 cases of Hb H disease, 5 cases of Hb H with Hb Constant Spring (CS), 9 cases of beta-thalassemia/Hb E disease and 1 case of beta-thalassemia major. Two cases were in Grade I, 10 cases in grade II, 7 cases in Grade III and one case in grade IV severity of DHF. Though there were evidences of plasma leakage, instead of hemoconcentration, eighteen patients (90 percent) had hematocrit dropped at the range of 11-66% of the initial level. Fifteen patients (75 percent) required at least one packed red cell transfusion. Nine patients (45 percent) had mild bleeding symptoms, one of them had upper gastrointestinal hemorrhage requiring platelet concentrate transfusion. Two patients (10 percent) had serious complications including one with infection-associated hemophagocytic syndrome (IAHS) requiring intravenous immunoglobulin (IVIG) and packed red cell transfusion and the other had generalized seizure due to hyponatremia and hypotension. No mortality was observed among this group of patients. Early recognition of the DHF in thalassemic patients and appropriate packed red cell transfusion in patients with anemic symptoms is warranted to reduce morbidity and mortality in these patients.
    Journal of the Medical Association of Thailand = Chotmaihet thangphaet 12/2005; 88 Suppl 8:S80-5.
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    ABSTRACT: Febrile neutropenia (FN) is a common and important clinical problem in pediatric cancer. Our Institution has developed a clinical practice guideline (CPG) for treatment of FN to assist the clinicians taking care of these patients. To evaluate characteristics of FN, sources and causative agents of infection, applicability and effectiveness of the CPG, and factors that associated with response to treatment. The medical records of patients with FN that had completed data from September, 2003 to May, 2005 were reviewed and analysed. A total of 148 FN episodes in 90 patients were analysed. The predominant underlying malignancy was acute leukemia. About 50% had absolute neutrophil count (ANC) less than 100 cells/mm3 at the beginning and at reassesment on day 3 of treatment. The causes of infection with microbiological confirmation was 25%. Urinary tract infection was the predominant source of infection and gram negative bacteria was the predominant causative agent. Sixty-two percents responded to initial treatment without changing of antibiotics. Of all episodes, 91.2% were able to complete treatment according to the CPG. The mortality rate was 1.4%. ANC of less than 100 cell/mm3 on day 3 of treatment was the significant risk factor for prolonged duration of fever and unresponsiveness to low risk regimen of antibiotics. ANC of less than 100 cell/mm3 on day 3, having hematologic malignancies, and recurrent fever were associated risks for the need for antifungal agent or referral to infectious diseases specialist or death. The pretreatment ANC more than 100 cells/mm3 was a significant predictor for the responsiveness to low risk regimen without recurrent fever. Our CPG could practically be applied in FN patients and resulted in low mortality rate.
    Journal of the Medical Association of Thailand = Chotmaihet thangphaet 12/2005; 88 Suppl 8:S124-34.
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    Kleebsabai Sanpakit
    Journal of the Medical Association of Thailand = Chotmaihet thangphaet 09/2005; 88(8):1147-52.
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    ABSTRACT: Acute haemolysis associated with mild upper respiratory tract infection was observed in a Thai boy who presented with a rapid decline in haemoglobin (Hb) levels, haemoglobinuria and evidence of intravascular haemolysis. Several possible causes giving rise to such a condition were excluded including G6PD deficiency, which is extremely common in Thailand. Subsequent haematological and molecular analyses demonstrated that the patient was homozygous for Hb Constant Spring (Hb CS/CS), an a globin haemoglobinopathy. It has been shown previously that patients with homozygous Hb CS had mild haemolytic anaemia secondary to an accumulation of alpha(CS) chains, which are toxic to red blood cell membrane cytoskeletons. Increased body temperature might induce more precipitation of this a globin variant. This report highlights the importance of Hb CS/CS as a potential predisposing cause of acute haemolysis in children that might be aggravated by acute bacterial or viral infections. This is particularly relevant for patients of Southeast Asian descent where this abnormal haemoglobin is highly prevalent.
    Annals of Tropical Paediatrics International Child Health 01/2005; 24(4):323-8. · 0.92 Impact Factor