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BACKGROUND Pleraxifor for peripheral blood stem cell (PBSC) mobilization in children with malignancies is often given following failure of standard mobilization (SM) rather than as a primary mobilizing agent. STUDY DESIGN AND METHODS In this retrospective multicenter study, we report the safety of plerixafor-based PBSC mobilization in children with malignancies and compare outcomes between patients who received plerixafor upfront with SM (Group A) with those who received plerixafor following failure of SM (Group B). In the latter pleraxifor was given either following a low peripheral blood (PB) CD34 (

Introduction In Europe and Northern America, the majority of children with high-risk neuroblastoma survive the disease. Elsewhere, treatment outcomes are poor. Methods A retrospective review of children treated for high-risk neuroblastoma in a single institution in Singapore from 2007 to 2019 was carried out. Treatment consisted of intensive chemotherapy, surgery aimed for gross total resection of residual disease after chemotherapy, consolidation with high-dose therapy followed by autologous stem cell rescue, radiotherapy to the primary and metastatic sites, followed by maintenance treatment with either cis-retinoic acid or anti-disialoganglioside (anti-GD2) monoclonoal antibody therapy. Survival data were examined on certain clinical and laboratory factors. Results There were 57 children with 32 males treated for high-risk neuroblastoma. Their mean age was 3.9 (0.7-14.9) years. The median follow-up time was 5.5 (1.8-13.0) years for the surviving patients. There were 31 survivors with 27 surviving in first remission, and 5-year overall survival and event-free survival rates were 52.5% and 47.4%, respectively. On log-rank testing, only the group of 17 patients who were exclusively treated at our centre had a survival advantage. Their 5-year overall survival rate, compared with others whose initial chemotherapy was done elsewhere, was 81.6% versus 41.1% (p = 0.011), and that of event-free survival was 69.7% versus 36.1% (p = 0.032). Published treatment results were found from four countries in Southeast Asia with 5-year overall survival rates from 13.5%-28.2%. Conclusion Intensified medical and surgical treatment for high-risk neuroblastoma proved to be effective with superior survival rates compared with previous data from Southeast Asia.

INTRODUCTION Childhood immune thrombocytopenia (ITP) remains a diagnosis of exclusion when isolated thrombocytopenia is not part of another disease process. In practice, the diagnosis of ITP can only be confirmed when thrombocytopenia resolves or is excluded after the recognition of a primary cause.

Manual examination of the peripheral blood smear (PBS) is currently performed on a fraction of samples sent for automated complete cell count. 39 children (age range 0-16.2 years) referred to a private paediatric practice during a 16-month period were retrospectively reviewed. Clinical scenarios, haematological features, laboratory-initiated PBS review, haematologist's PBS review and final diagnosis were described. Clinical indications included isolated thrombocytopenia (n = 10), unexplained bruises (n = 5), acute febrile illnesses (n = 11), anaemia (n = 8) and others (n = 5). The laboratory reviewed the PBS in 30 cases according to preset criteria and made no conclusive remarks. All slides were reviewed by a haematologist and a diagnosis was made in 27 (69%) cases, including 7 (78%) of the nine slides the laboratory did not review. The practice of laboratory-initiated PBS review requires re-evaluation. Haematologist-reviewed PBS is an important diagnostic tool for children with anaemia, bleeding disorders and acute febrile illnesses.

Acute pancreatitis is a rare presentation in Burkitt lymphoma (BL) and may lead to delayed medical or unnecessary surgical treatment. Three cases of BL presenting as acute pancreatitis in the authors' institutions were described. Similar cases reported in the medical literature were collected and described along with the authors' cases. There were 12 cases described in the medical literature and hence a total of 15 cases of BL presenting as acute pancreatitis. Fourteen cases were the first diagnosis, and the other presented at lymphoma relapse. Twelve cases occurred in children under 15 years. Twelve patients had extrapancreatic disease. Three children were treated with surgery before diagnosis. Two patients died. Six of the remaining had adequate follow-up and were surviving in remission 8 months to 16 years after diagnosis. Lymphoma should be included in the differential diagnosis of acute pancreatitis in children. Acute pancreatitis in combination with malignant infiltration on imaging is highly suggestive of BL, especially in the jaundiced child.

Macropolycytes are tetraploid neutrophils produced during accelerated myelopoiesis. They have been reported in adults with pernicious anemia, sepsis, and after cytotoxic chemotherapy. Two pediatric cases are reported, one after granulocyte colony-stimulating factor treatment and the other following Kawasaki disease, respectively.

An acquired, transient bleeding disorder that predominantly affects children in Southeast Asia has been reported for the last 4 decades. The condition has been named idiopathic purpura with gray platelets (IPGP) or acquired platelet dysfunction with eosinophilia. In a retrospective review from a private pediatric clinic over an 8-year period, 10 consecutive children were diagnosed as IPGP with a mean age of 8.4 (3.7 to 16.2) years. Eosinophilia (>0.5×10/L) was absent in 1, while gray platelets were consistently found in all cases with a mean proportion of 64.5% (40% to 80%). Platelet aggregation tests were performed in 9 patients with abnormal responses consistent with platelet storage pool defect. All children recovered completely and spontaneously from 1 to 4 months after diagnosis without specific therapy. In an otherwise well child who presents abruptly with easy bruising and a platelet count >100×10/L, IPGP can be readily recognized as an acquired form of gray platelet syndrome. Eosinophilia is common but not mandatory for diagnosis.

In our institution, hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) has been treated with N-acetylcysteine (NAC) since 2008-a loading dose of 150 mg/kg, followed by 12 doses of 70 mg/kg 6 hourly. Nine children were diagnosed with hepatic VOD/SOS. Hepatic VOD/SOS occurred in seven children after stem cell transplantation and two were receiving chemotherapy for Wilms tumor. Their clinical severity was classified as moderate in two and severe in seven by the European Society for Blood and Marrow Transplantation criteria. All children recovered and were discharged from 4 to 16 days after diagnosis. No side effects were observed. Intravenous NAC is an effective treatment for hepatic VOD/SOS.

Hepatic sinusoidal obstruction syndrome is commonly described in pediatric oncology as a complication of chemotherapy. It has also been occasionally reported in adult cancer patients. Treatment is largely supportive with fluid restriction. A 16-month-old girl with stage II Wilms tumor receiving post-nephrectomy chemotherapy with dactinomycin and vincristine developed hepatic sinusoidal obstruction syndrome with painful hepatomegaly, ascites with significant weight gain, grossly deranged liver function, severe thrombocytopenia, and reversal of blood flow in the portal vein on Doppler sonography. Treatment with N-acetylcysteine was followed by complete resolution of clinical signs and amelioration of laboratory abnormalities within 72 hours of treatment. N-acetylcysteine is a safe and probably an effective treatment for dactinomycin-induced hepatic sinusoidal obstructive syndrome.

Severe combined immunodeficiencies (SCID) are a group of rare inherited disorders with profound defects in T cell and B cell immunity. From 2005 to 2010, our unit performed testing for IL2RG, JAK3, IL7R, RAG1, RAG2, DCLRE1C, LIG4, AK2, and ZAP70 mutations in 42 Chinese and Southeast Asian infants with SCID adopting a candidate gene approach, based on patient's gender, immune phenotype, and inheritance pattern. Mutations were identified in 26 patients, including IL2RG (n = 19), IL7R (n = 2), JAK3 (n = 2), RAG1 (n = 1), RAG2 (n = 1), and DCLRE1C (n = 1). Among 12 patients who underwent hematopoietic stem cell transplantation, eight patients survived. Complications and morbidities during transplant period were significant, especially disseminated bacillus Calmette-Guérin disease which was often difficult to control. This is the first cohort study on SCID in the Chinese and Southeast Asian population, based on a multi-centered collaborative research network. The foremost issue is service provision for early detection, diagnosis, management, and definitive treatment for patients with SCID. National management guidelines for SCID should be established, and research into an efficient platform for genetic diagnosis is needed.

In this cross-sectional study, we compared the quality of life (QOL) in transfusion-dependent thalassemic patients who survived matched sibling hematopoietic SCT (HSCT, n=24) with patients treated conventionally with transfusion and iron chelation (n=74). WHOQOL-BREF(HK) and PedsQL questionnaires were administered to patients aged >18 years and 5–12 years, respectively. Patients aged 12–18 years received both questionnaires. WHOQOL-BREF(HK) revealed post transplant patients rated overall health better than those treated conventionally (score 3.67 vs 3.06, P=0.01). They are less dependent on medical aids (3.87 vs 2.96, P=0.006), having higher activity level (4.00 vs 3.36, P=0.026) and better personal relationships (4.13 vs 3.69, P=0.014). Physical health domain score was better (75.20 vs 63.94, P=0.007). These differences remained significant after adjustment for comorbidities. PedsQL revealed post transplant patients rated better for running (3.53 vs 2.72, P=0.001) and sports (3.20 vs 2.64, P=0.038), even after adjustment for comorbidities, but were less satisfied for school absence to attend hospital (2.53 vs 3.29, P=0.03). Post transplant patients were significantly more likely to consider marriage (100 vs 75.7%, P=0.033), but not childbearing (66.7 vs 51.4%, P=0.28). In conclusion, transplanted thalassemic patients enjoy better QOL, mainly in physical health, compared with conventionally treated patients. This information is important to patients considering HSCT.

Enhanced fetal hemoglobin (Hb F) production can partially compensate for the lack of adult hemoglobin (Hb A) in patients with β-thalassemia major or intermedia, and ameliorate the clinical severity of these diseases. To further elucidate factors governing Hb F levels, we evaluated demographic, clinical, laboratory, and genetic characteristics in 241 unrelated adult β-thalassemia carriers in Hong Kong. They had wide variations in Hb F and F-cell numbers skewing toward higher levels. Individuals who coinherited the Xmn IT-allele in the Gγ-globin gene promoter had higher Hb F and more F-cells compared with those lacking the Xmn I T-allele. However, both groups exhibited a similarly wide spread of Hb F and F-cells. The correlation of Hb F and F-cells corresponded well to both linear and exponential models, suggesting multiple mechanisms for Hb F augmentation. The heritabilities of Hb F and F-cells were calculated in 66 families (111 parents who were β-thalassemia carriers and 82 asymptomatic offspring) to be 0.7 to 0.9. The Xmn I polymorphism accounted for 9% of the Hb F and 13% of the F-cell heritabilities. These results suggest that these family members are well suited for genome wide association studies that will identify genetic loci regulating Hb F production, and likely novel pharmacological targets for reactivating Hb F production in adults. Am. J. Hematol., 2008. © 2008 Wiley-Liss, Inc.

An extended family with three individuals affected by two different forms of double heterozygosity for beta-thalassemia and Hb New York is reported. Double heterozygosity of Hb New York [beta 113 GTG-->GAG; VAL-->GLU] and beta degrees codon 17 was detected in a fetus following prenatal screening for thalassemia. The father and a paternal aunt were also found to be heterozygous for Hb New York and beta degrees IVSII-654. Both adults had clinical and hematological features consistent with beta-thalassemia trait. The affected child was followed up after birth and manifested the typical course of a thalassemia trait, with no signs of organomegaly or overt hemolysis. Observations strongly suggest that double heterozygosity of Hb New York and beta degrees thalassemia has mild, if any, clinical symptoms, and is not an indication of therapeutic abortion when detected antenatally.

Two cases of spinal epidural hematoma and two cases of intracranial subdural hematoma after lumbar puncture (LP) are reported in children receiving chemotherapy for acute lymphoblastic leukemia and non-Hodgkin lymphoma. The bleeding was asymptomatic but interfered with treatment in one case, and caused either severe backache or headache but no neurological deficit in the other three patients. The platelet counts were 8 and 46 × 109/L in two patients and were normal in the other patients at the time of LP. All recovered without surgical treatment. There is an inherent, albeit uncommon, risk of bleeding into the central nervous system associated with LP in children with cancer and should be distinguished from postdural puncture headache (PDPH). Thrombocytopenia is not always an accompanying factor. Pediatr Blood Cancer 2007;48:233–237. © 2006 Wiley-Liss, Inc.

Subcutaneously tunneled, cuffed central venous catheters (CVCs) are commonly used in children undergoing cytotoxic chemotherapy or hematopoietic stem-cell transplantation. When their use is no longer indicated or precluded by mechanical or infectious complications, CVCs have to be removed. General instructions on how cuffed CVC should be removed are available in the medical texts but none is adapted for use in children. A literature search from the MEDLINE and EMBASE to identify articles describing the procedure of removing CVC or complications arising from the procedure was carried out. Specific guidance on the removal of CVC in children was not found. Venous air embolism appeared to be the most common complication associated with catheter removal but none involved pediatric patients. On the other hand, three out of the five incidents of catheter fracture with or without embolization happened in children. Further studies are needed to define the optimal management of CVC removal in pediatric patients. A sequence of positioning the child, use of sedation, dissecting out the cuff, pulling off the catheter, closing the exit wound, and handling of the removed catheter is suggested.

To study the outcomes of children hospitalized for suspected child abuse before and after the implementation of a management protocol in a hospital in Hong Kong.Two 2-year periods before (1994-1995) and after (2002-2003) the implementation of the protocol in 1998.This is a retrospective hospital chart review in which the patients' characteristics, the use of laboratory and radiological examination, abuse substantiation and official registrations are compared between the two study periods.There were 109 and 320 patients admitted for evaluation of child abuse for the periods 1994-1995 and 2002-2003, respectively. Children in both periods were similar in sex ratio, proportion of severe forms of child abuse, rates of abuse substantiation and inclusion in the Child Protection Registry. After the implementation of a management protocol, there has been a significant drop in the proportion of children subjected to investigations such as blood counts (86% vs. 16%, p.001), clotting study (75% vs. 9%, p.001), and skeletal survey (78% vs. 6%, p.001). The average length of hospital stay also dropped from 15.3 days to 6.1 days (p.001).There has been an almost threefold rise in the number of child abuse cases handled at the hospital during the 10-year interval. With the implementation of a management protocol, only a small proportion of children need laboratory investigations or skeletal survey without any drop in abuse substantiation and official registration. The length of hospital stay has also been significantly reduced.

A 17-year-old boy with acute lymphoblastic leukemia developed acute renal failure within 48 h of an intravenous high-dose methotrexate (5 g/m2) infusion. His renal function returned to baseline 14 days later with supportive care, folinic acid rescue, and urinary alkalinization. A retrospective review revealed that the patient had been exposed to iopamidol, an intravenous contrast medium, on the day prior to the commencement of methotrexate treatment. Methotrexate-associated nephropathy is a rare complication in pediatric oncology, and a review of the literature suggests that exposure to nephrotoxic agents may be a significant but perhaps underrecognized risk factor for its development.

A controlled, open-label and randomized study was conducted to evaluate the safety and efficacy of the oral iron chelator deferiprone (L1) in thalassemia major patients from Hong Kong. Forty-nine patients were recruited in total (median age: 20 years; range: 8 to 40 years). The division of the patients was determined based on liver iron content and put into either the poorly-chelated (Group I) or well-chelated (Group II) groups. In Group I, 20 patients received combined therapy of L1 daily plus desferrioxamine (DFO), in a reduced frequency of twice weekly, while the control group consisted of 16 patients who were treated with DFO alone. In Group II, six patients received L1 only, while the control group consisted of seven patients treated with DFO alone. Only patients who participated for longer than 6 months were analyzed for efficacy (n = 44). The median study period was 18 months. Transient and mild gastrointestinal upset (31%), joint pain (15%) and liver enzyme elevation (23%) were the most common side effects noted for L1. No case of neutropenia was observed in this study. Serum ferritin (SF) levels showed significant decline in the poorly-chelated patients using combined therapy (L1 and reduced frequency DFO) as compared to those on DFO alone. However, their pre- and post-study liver iron content was not significantly different. Evaluation of the well-chelated group demonstrated no significant change in SF or liver iron content in both the study and control arms. We conclude that the short-term use of L1, with or without DFO, was safe and efficacious in our Chinese patient cohort. The long-term efficacy of reducing iron overload by treatment regimens including L1 requires further study.

Multidisciplinary case conference is an important component in the management of suspected cases of child abuse and neglect in Hong Kong. The medical practitioner is one of the key professionals in the case conference to discuss the nature of the case, issues concerning child protection, and interventions to safeguard the child's subsequent welfare interests. Yet, specific guidance for the medical profession is lacking. In this article, the authors present an overview of the child protection case conference and practical suggestions for the medical practitioner when called to participate in the multidisciplinary case conference.

There have been two previous reports on Hb Val de Marne (Hb Footscray) [alpha133(H16)Ser--Arg] in the literature, but the molecular characterization has hitherto not been described. Based on the Ser--Arg transition, the presumed mutation was cited as AGC--CGC of the alpha2- or alpha1-globin gene. We have found this variant in a 15-year-old Chinese girl and her father, and automated DNA sequencing revealed an AGC--AGA mutation at codon 133 of the alpha2-globin gene. Since an increasing number of alpha-globin gene variants have been reported with the same protein alteration but with different mutations on the alpha1- or alpha2-globin genes, the mutation identified in the present family does not preclude the presence of other alpha-globin gene mutations leading to this hemoglobin (Hb) variant.

The authors report a case of thrombocytopenia in a 10-year-old Chinese girl who was also found to have Graves disease 6 months later. The clinical, hematologic, and bone marrow features were indistinguishable from chronic idiopathic thrombocytopenic purpura, but the patient's platelet counts were normalized concurrently with the control of thyrotoxicosis with carbimazole treatment. Childhood thrombocytopenia associated with autoimmune thyroid disease is a rarely reported condition and its management differs from that of idiopathic thrombocytopenic purpura.

Inferior vena caval (IVC) filter thrombosis in patients with contraindications to anticoagulant therapy is a difficult and challenging clinical problem. We report our experience in treating three such patients using a mechanical thrombectomy device, which resulted in rapid symptomatic relief until anticoagulant therapy could be safely introduced.

A 2-year-old girl was referred for the management of the progression of her localized Wilms tumor. She had undergone laparoscopic nephron-sparing surgery for her right Wilms tumor 3 months earlier. Postoperative chemotherapy was commenced 6 weeks later. Subsequent computer tomographic evaluation 12 weeks postsurgery showed local recurrence and peritoneal tumors. These findings were confirmed at laparotomy. We report the first case of peritoneal tumor dissemination after laparoscopic nephron-sparing surgery for localized Wilms tumor.

Background Bilateral ovarian Burkitt's lymphoma was diagnosed in a 10-year-old girl who presented with abdominal pain, constipation, and signs of ovarian torsion. Case Left salpingo-oopherectomy and biopsy of the right ovary were carried out as initial surgical intervention. She remained well in first complete remission at 25 months after multiagent chemotherapy, with pubertal development on clinical examination, hormonal measurements, and pelvic sonography. Summary and Conclusion Although rare, Burkitt's lymphoma should be included in the differential diagnosis of bilateral ovarian tumors in childhood, and more conservative surgical management is indicated to preserve ovarian function and fertility.

The thalassemias are common in Hong Kong. One of the severe forms, beta-thalassemia major, has been preventable locally by antenatal screening since 1983. Yet new patients are still being diagnosed. In this retrospective study, 34 children with severe beta-thalassemia syndromes were diagnosed in two major public hospitals between 1990 and 1996. They included one pair of identical twins and two pairs of siblings. Twenty-seven (79%) had homozygous beta-thalassemia and seven (21%) had beta E thalassemia. All but four (12%) were transfusion dependent. Fifty-five (89%) parents had been evaluated for their thalassemia status. Forty-eight had beta-thalassemia traits and seven were hemoglobin E carriers. The reasons for the birth of these children with severe beta-thalassemia syndromes were (1) late or no antenatal visit (n = 8, 24.2%), including three cross-border deliveries in which the pregnant mothers came from mainland China to Hong Kong for confinement, (2) lack of maternal screening (n = 13, 39.4%), (3) lack of paternal screening (n = 7, 21.2%), (4) parental refusal (n = 3, 9.1%), and (5) unknown (n = 2, 6.1%). These findings suggest that several factors undermine the effectiveness of antenatal screening for prevention of thalassemias. Many medical practitioners and the general public are still not aware of the screening procedures. The migration of population from mainland China to Hong Kong may result in the birth of many more children with beta-thalassemia major.

A case of multifocal malignant peripheral neuroectodermal tumor (PNET) arising from a plexiform neurofibroma in a 4-month-old Chinese boy with neurofibromatosis type 1 (NF-1) is described. Cytogenetic culture demonstrated hypotriploid karyotype with an abnormal clone characterized by 59–60, XY, +2, +3, +6, +8, +8, +12, +i(13)(q10), +der(14)t(1;14)(q21;q32), +16, +19, +20, +mar[cp3] with no apparent abnormality of chromosome 17. The child was treated with combination chemotherapy comprising ifosphamide, vincristine and doxorubicin. Despite initial partial response the child finally died of tumor progression and pulmonary metastases 8 months after diagnosis. We believe this is the first reported case of PNET in a child with NF-1 and may support an association between these two disorders of neural crest origin. © 1996 Wiley-Liss, Inc.

A 19-year-old girl with thalassemia intermedia presented with signs of thoracic spinal cord compression secondary to extramedullary hematopoiesis. She was started on a transfusion regimen to maintain a hemoglobin level of more than 12.5 g/dL. Clinical signs disappeared within the first week and circulating erythroblastemia was completely suppressed by the second week. Magnetic resonance imaging 4 weeks after diagnosis revealed near-complete resolution of the extradural mass, followed by gradual improvement in the posterior tibial somatosensory evoked potentials. Transfusion therapy may be diagnostically and therapeutically useful in spinal cord compression secondary to extramedullary hematopoiesis, obviating the need for surgery or radiotherapy.

A 13-year-old girl with Plesiomonas shigelloides septicemia is reported. The infection occurred while she was receiving an allogeneic bone marrow transplantation for acute promyelocytic leukemia. Treatment with ciprofloxacin was successful. Twenty-one cases of Plesiomonas septicemia have been reported in the literature. Immunocompromised hosts, especially neonates, are commonly affected. The case mortality rate is high, with 13 of the reported patients dying of the infection. Successful treatment relies on the early identification of the organism and implementation of effective antibiotics.

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.

pp. 251-317 of this journal issue entitled: Abstracts Presented for the 1995 International Symposium on Clinical Immunology

Methodology: A cross-sectional study of growth, puberty and endocrine function was performed on 35 girls and 33 boys with thalassaemia major. Results Despite regular transfusion and chelation therapy, 75% of the girls and 62% of the boys over the age of 12 years were below the third percentile for height. Hypogonadotropic hypogonadism was found in a similar percentage of patients. Moderate to marked zinc deficiency secondary to chelation therapy was considered unlikely because normal serum zinc levels were found in all but three of our patients, but we could not exclude the possibility of a marginal status of zinc nutrition causing growth failure. Growth hormone deficiency and diabetes mellitus were sometimes encountered but hypothyroidism, hypoparathyroidism and adrenal insufficiency were rare among our patients. Most of the patients with growth failure had normal growth hormone (GH) response to insulin induced hypoglycaemia. The serum insulin-like growth factor-1 (IGF-1) levels were low in our patients and no significant difference in the serum IGF-1 levels was found between prepubertal children with or without growth failure (0.4±0.1 mU/mL vs 0.37±0.11 mU/mL, P = 0.39). Similarly, no difference in the serum IGF-1 levels was found between pubertal children with or without growth failure (0.48 ± 0.2 U/mL vs 0.56 ±0.14 U/mL, P= 0.26). Conclusions Delayed sexual maturation and a possible defect in growth unrelated to the GH-IGF-1 axis may be responsible for the growth failure in adolescent children with thalassaemia major.