Search

Polycystic ovary syndrome (PCOS) is a touchy clinical and public health problem worldwide, which adversely affects women's health and health-related comorbidities for lifetime, and represents a tremendous burden for both the family of the patient and for society. According to the diagnostic criteria used and the population studied, the prevalence rate of PCOS is between 6% and 21%. However, current conventional modern medicines for PCOS are only moderately effective at controlling the signs and symptoms, while they are not thoroughly able to prevent complications. Therefore, many patients have turned to complementary and alternative medical (CAM) treatments. CAM use is wide spread among patients with PCOS, and more than 70% of patients use CAM at one point during their diseases. The patients' primary motivations include dissatisfaction with available medications, perceive higher risk of drug side effects and crushing health burden and economic costs, desire for symptom relief, pursuit of shortening the course of disease, and the belief that CAM therapy is in accordance with the patients' values and beliefs. At present, several CAM methods have been used in women with PCOS, which has achieved obvious effects. However, biologically plausible mechanisms of the action of traditional Chinese medicine- (TCM-) associated CAM for PCOS have not been systematically reviewed. This review briefly summarizes the current progress of the impact of herbal medicine on the outcomes of PCOS and introduces the mechanisms. [ABSTRACT FROM AUTHOR]

Background: Most mammary Paget disease (MPD) is associated with underlying in situ or invasive breast cancer. The objective of this study was to compare the clinicopathological characteristics and survival outcomes between breast cancer with Paget disease (PD) and breast cancer alone. Methods: From the Surveillance, Epidemiology, and End Results (SEER) database, 2000‐2015, of the US National Cancer Institute, we identified 1569 women who had PD with invasive ductal carcinoma (PD‐IDC) and 1489 women who had PD with ductal carcinoma in situ (PD‐DCIS). Independent demographic and clinicopathological variables as well as survival outcomes of these patients were compared to patients with the corresponding breast cancer without concomitant PD. Results: PD‐IDC and PD‐DCIS both had worse survival outcomes and poorer tumor characteristics than the corresponding disease without PD. Contrary to in the breast cancer alone groups, in the breast cancer with PD groups, the HR status (P = 0.182 in PD‐IDC and P = 0.371 in PD‐DCIS), HER2 status (P = 0.788 in PD‐IDC and P = 0.643 in PD‐DCIS), and combined molecular subtype (P = 0.196 in PD‐IDC and P = 0.853 in PD‐DCIS) were not found to affect disease prognosis. After matching tumor characteristics and treatment approaches, PD‐IDC as well as PD‐DCIS exhibited no significant difference in disease prognosis with corresponding IDC and DCIS. Finally, by comparative analysis, a kind of PD‐DCIS (ICD‐O‐3 code 8543/3) showed many invasive behaviors (31.8% of 8543/3 patients had stage I‐III cancer) and was associated with worse survival outcomes than the other type of PD‐DCIS. Conclusions: Breast cancer with concomitant PD was associated with more aggressive tumor characteristics and worse survival outcomes. The HR status, HER2 status, and combined molecular subtype could not affect the prognosis of breast cancer with PD. Moreover, a portion of the PD‐DCIS cases were invasive breast cancer cases that required special treatment. [ABSTRACT FROM AUTHOR]

Abstract: Verinurad (RDEA3170) is a selective uric acid reabsorption inhibitor in clinical development for treatment of hyperuricemia and gout. This phase 1b, multiple‐dose, drug‐drug interaction study evaluated the pharmacokinetics, pharmacodynamics, and tolerability of verinurad in combination with allopurinol. Adult males with gout were randomized to receive once‐daily oral doses of allopurinol 300 mg or verinurad 10 mg alone for 7 days, allopurinol 300 mg + verinurad 10 mg on days 8 to 14, and the alternative single agent on days 15 to 21. Colchicine 0.6 mg was taken prophylactically for gout flares. Plasma/serum and urine samples were assayed for verinurad, allopurinol, oxypurinol (allopurinol active metabolite), colchicine (plasma only), and uric acid. Safety was assessed by adverse events (AEs) and laboratory tests. Verinurad plasma exposure was unaffected by allopurinol. Verinurad increased the maximum observed plasma concentration (Cmax) for allopurinol by 33%; the area under the plasma concentration‐time curve (AUC) was unaffected. Oxypurinol Cmax and AUC were reduced 32% and 38%, respectively, by verinurad. Colchicine plasma exposure was unaltered by verinurad. The maximum decrease in serum urate was greater with verinurad + allopurinol (65%) than with verinurad (51%) or allopurinol (43%) alone. Compared with the baseline rate, the maximum rate of uric acid excreted in urine was +56% with verinurad, −46% with allopurinol, and unchanged with verinurad + allopurinol. No serious AEs, discontinuations due to AEs, or clinically significant laboratory abnormalities were noted. Despite decreased systemic exposure of allopurinol and oxypurinol in the presence of verinurad, the combination resulted in greater serum urate reduction compared with either drug alone and was well tolerated at the studied doses. [ABSTRACT FROM AUTHOR]

The present study aimed to determine whether the expression of microRNA (miR)-10b was correlated with the molecular subtypes of early invasive ductal carcinoma of the breast. In situ hybridization was used to detect the expression of miR‑10b in 193 patients diagnosed with early invasive ductal carcinoma. Immunohistochemistry was performed to evaluate the expression of estrogen receptor (ER)‑α, progesterone receptor (PR) and human epidermal growth factor receptor‑2 (Her‑2). The positive expression rate of miR‑10b in patients with early invasive ductal carcinoma with ER‑α (+) or PR (+) was decreased compared with ER‑α (‑) or PR (‑) patients (P<0.05). Furthermore, the positive expression rate of miR‑10b in patients with Her‑2 (‑) was significantly increased compared with patients that were Her‑2 (+) (P=0.031). The positive expression rate of miR‑10b in the luminal B subtype was significantly decreased compared with that in the luminal A, Her‑2 and basal‑like subtypes (P=0.037). In patients that were identified as miR‑10b (+), the median disease‑free survival time was significantly increased in patients that were ER‑α (+)/PR (+)/Her‑2 (‑) compared with patients that were ER‑α (‑)/PR (‑)/Her‑2 (+) (P<0.05). In addition, the median disease‑free survival time was significantly decreased in Her‑2 overexpression and basal‑like subtypes when compared with luminal A and B subtypes (P<0.05). The molecular subtype was an independent prognostic factor for early invasive ductal carcinoma (odds ratios for luminal B, Basal‑like, and Her‑2 overexpression were 2.900, 5.232 and 4.214, respectively; all P<0.05). Positive expression of miR‑10b may also be a prognostic risk factor (odds ratio >1), though this was not statistically significant (P>0.05). The present findings indicated that miR‑10b‑positive expression was correlated with the expression of ER‑α, Her‑2 and the molecular subtypes of early invasive ductal carcinoma of the breast. [ABSTRACT FROM AUTHOR]

Objective: Our objectives were to determine whether a change in serum uric acid (sUA) resulted in a corresponding change in the fractional excretion of uric acid (FEUA) and whether the renal response was different in patients with gout versus healthy subjects.Methods: FEUA was calculated from previously published studies and four new phase I studies in healthy subjects and/or patients with gout before and after treatment to lower or raise sUA. Treatments included xanthine oxidase inhibitors to lower sUA as well as infusion of uric acid and provision of a high-purine diet to raise sUA. Plots were created of FEUA versus sUA before and after treatment. For the phase I studies, percent change in FEUA per mg/dL change in sUA was calculated separately for healthy subjects and patients with gout, and compared using Student's t test.Results: Analysis of previously published data and the new phase I clinical data indicates that changing sUA by a non-renal mechanism leads to a change in FEUA. The magnitude of change is greater in subjects with higher baseline FEUA versus patients with gout. Healthy subjects excrete more urate than do patients with gout at physiological urate-filtered load; this difference disappears when the urate-filtered load is decreased to ∼5000mg/24hours.Conclusion: These observations are consistent with a less saturated urate reabsorption system in patients with gout versus healthy subjects, resulting in elevated retention of uric acid. Further investigation could lead to the discovery of mechanisms responsible for the etiology of hyperuricemia/gout. [ABSTRACT FROM AUTHOR]

Paeoniflorin (PF) is the main active component extracted from the roots of Paeonialactiflora, a traditional Chinese medicine used for the treatment of neurodegenerative disorders, especially Parkinson's disease (PD). The degeneration of dopaminergic (DA-) neurons in PD may be caused by pathological activation of acid-sensing ion channels (ASICs). Thus, we designed a series of experiments to evaluate the therapeutic effects of PF and to test whether its effects are related to its inhibitory effect on ASIC1a. We found that systemic administration of PF or ASICs blockers (psalmotoxin-1 and amiloride) improved behavioral symptoms, delayed DA-neuronal loss and attenuated the reduction of dopamine (DA) and its metabolites in a rat model of 6-hydroxydopamine (6-OHDA)-induced PD. In addition, our data showed that PF, like ASICs blockers, regulated the expression of ASIC1a, decreased the level of α-synuclein (α-SYN), and improved autophagic dysfunction. Further experiments showed that ASIC1a knockdown down-regulated the α-SYN level and alleviated the autophagic injury in the 6-OHDA-treated ASIC1a-silenced PC12 cells. In summary, these findings indicate that PF enhanced the autophagic degradation of α-SYN and, thus, protected DA-neurons against the neurotoxicity caused by 6-OHDA. These findings also provide experimental evidence that PF may be a neuroprotectant for PD by acting on ASIC1a and that ASIC1a may be involved in the pathogenesis of PD. [ABSTRACT FROM AUTHOR]

Background: Neurosyphilis is caused by the invasion of Treponema pallidum into the central nervous system. General paresis (GP) is a type of neurosyphilis. The main manifestation of general paresis is dementia; however, this is different from the other types of dementia, which can be cured by adequate doses of penicillin in the early stage. Neurosyphilis is the "great imitator" because it can mimic many types of medical disorders. In addition, the manifestations of neurosyphilis are not typical. Psychiatric disorders as a cause of general paresis have become more common due to the use of antibiotics. Patients with a psychiatric manifestation are often misdiagnosed. The purpose of this study was to explore the differences in the clinical and neuropsychological characteristics of general paresis between patients misdiagnosed as having a primary psychiatric disease and patients diagnosed correctly upon seeing a doctor. The results may assist clinicians in the early identification of neurosyphilis with a mental disorder. Method: The demographic and clinical manifestations, laboratory tests, and neuroimaging and neuropsychological characteristics were analysed in 55 general paresis patients with psychiatric disorders, including 29 patients misdiagnosed as primary psychiatric disease and 26 patients diagnosed as having general paresis after being seen once by a doctor. Result: All of the patients had positive assay results for cerebral spinal fluid (CSF) Treponema pallidum hemagglutination (TPHA). Only 43.3 % of misdiagnosed patients and 30.8 % of general paresis patients had positive results for the CSF rapid plasma reagin (RPR) test; 96.4 % patients had abnormal neuroimaging. Mood disturbances were the most common psychiatric disorder in the general paresis patients, especially agitation, between the two groups (patients with general paresis who were misdiagnosed as having primary psychiatric disease and patients who had never been misdiagnosed) (p = 0.011). Conclusion: Our findings reinforce the importance of performing serologic testing for syphilis. This should be a part of the evaluation of patients with psychiatric disorders, especially patients with cognitive impairment. When the syphilis serology is positive, the patient should be examined thoroughly for neurosyphilis by lumbar puncture. Brain imaging could also aid the physician in discriminating these patients from those with a functional mental disorder. [ABSTRACT FROM AUTHOR]

Alzheimer’s disease, one of the neurodegenerative diseases, shows the progressive senescence of neural progenitor/stem cells. N-Stearoyl-L-tyrosine (NsTyr) showed neuroprotective effect against chronic brain ischemia in previous reports. In the present study, we find the antisenescent effects of NsTyr-2K in NSPCs induced by Aβ1–42 in vitro. Cell viability of NSPCs was evaluated by CCK8 assay; SA-β-gal staining was used to evaluate senescence of NSPCs. CB receptors were detected by immunohistochemistry in NSPCs. AM251 or AM630 was used to offset the anti-senescence effects afforded by NsTyr-2K. The positive rate of SA-β-gal staining was significantly increased in NSPCs after incubation with Aβ1–42 for 9 days. CB receptors were found on the surface of NSPCs. The expression level of CB1 receptors was significantly decreased in NSPCs after incubation with Aβ1–42. This phenomenon was reversed dose-dependently by NsTyr-2K. NsTyr-2K attenuated Aβ1–42 induced NSPCs senescence dose-dependently, and its antisenescence effect was completely abolished by AM630. Aβ1–42 dose-dependently increased the prosenescence molecules p16 and Rb. Their expression was inhibited by NsTyr-2K dose-dependently and blocked by AM630 in NSPCs. These results suggest that NsTyr-2K can alleviate the senescence of NSPCs induced by Aβ1–42 via CB2 receptor. [ABSTRACT FROM AUTHOR]

Co-administration of an inhaled corticosteroid and long acting beta agonist for chronic obstructive pulmonary disease has reduced mortality compared to either drug alone. This combination reduces exacerbations, hospitalization, emergency department visits and health care costs. A novel fixed-dose combination of the long acting beta-2 agonist salmeterol xinafoate (SX) and the corticosteroid mometasone furoate (MF) were prepared in a composite particle formulation as brittle matrix powder (BMP) and investigated for suitability as an inhaled combination product. In this study, BMP fixed dose combinations of SX and MF with or without stabilizing excipients (lactose, mannitol, glycine and trehalose) were prepared and characterized with respect to their thermal properties, morphology, aerodynamic performance and physical stability. BMP combination formulations of SX and MF exhibited improved aerodynamic properties when delivered by dry powder inhalation as compared to the micronized blends of the same substances. Aerodynamic evaluation was carried out by next generation pharmaceutical impactor (NGI) with a marketed DPI device. Results demonstrated that co-deposition occurred when SX and MF were formulated together as composite particles in a BMP, while physical blends resulted in inconsistent deposition and dose uniformity. As a result of the bottom-up particle engineering approach, combination BMP formulations allow for dual API composite formulations to be dispersed as aerosolized particles. Aerosolized BMP combination formulations resulted in delivered dose uniformity and co-deposition of each API. Further, an excipient-free formulation, BMP SXMF, delivered approximately 50% of the loaded dose in the respirable range and demonstrated stability at ambient conditions for 6 months. Single dose 24-h pharmacokinetic studies in rats demonstrated that lung tissue deposition and blood circulation (AUC 0–24h ) of two APIs were higher for the BMP combination group exhibiting a significantly higher lung concentration of drugs than for the crystalline physical blend. While high system drug levels are generally undesirable in lung targeted therapies, high blood levels in this rodent study could be indicative of increased pulmonary tissue exposure using BMP formulations. [ABSTRACT FROM AUTHOR]

Alzheimer's disease (AD) and osteoporosis are two closely related multifactorial progressively degenerative diseases that predominantly affect aged people. These two diseases share many common risk factors, including old age, being female, smoking, excessive drinking, low estrogen, and vitamin D3 levels. Additionally, oxidative damage and the dysfunction of the antioxidant system play important roles in the pathogenesis of osteoporosis and AD. Aβ not only leads to impaired memory but also plays a crucial role in the demineralization process of bone tissues of older people and women with menopause. Curculigoside can promote calcium deposition and increase the levels of ALP and Runx2 in osteoblasts under oxidative stress via anti-oxidative character. Therefore, we investigated the effects of CUR on the spatial learning and memory by the Morris water maze and brain immunohistochemistry, and bone microstructure and material properties of femurs by micro-computed tomography and mechanical testing in APP/PS1 mutated transgenic mice. Oral administration of CUR can significantly enhance learning performance and ameliorate bone loss in APP/PS1 mutated transgenic mice, and the mechanism may be related to its antioxidant effect. Based on these results, CUR has real potential as a new natural resource for developing medicines or dietary supplements for the prevention and treatment of the two closely linked multifactorial progressive degenerative disorders, AD and osteoporosis. [ABSTRACT FROM AUTHOR]

The aim of this study is to survey and compare the development of auditory skills in young children with Mondini dysplasia and profoundly-deaf young children with radiologically normal inner ears over a period of 3 years after cochlear implantation. A total of 545 young children (age 7 to 36 months) with prelingual, severe to profound hearing loss participated in this study. All children received cochlear implantation. Based on whether or not there was a Mondini dysplasia as diagnosed with CT scanning, the subjects were divided into 2 groups: (A) 514 young children with radiologically normal inner ears and (B) 31 young children with Mondini dysplasia. The Infant-Toddler Meaningful Auditory Integration Scale (IT-MAIS) was used to assess the children's auditory skills that include vocalization changes, spontaneous alerting to sounds in everyday living environments, and the ability to derive meaning from sounds. The assessment was performed prior to surgery and at 1, 3, 6, 9, 12, 24, and 36 months after implant device switch-on. The mean scores for overall auditory skills were not significantly different between groups A and B at pre-surgery, 1, 12, 24, and 36 months post-surgery, but were significantly different at 3, 6, and 9 months post-surgery. The mean scores for all auditory skills in children with Mondini dysplasia showed significant improvement over time. The mean scores for the three subcategories of auditory skills in children with Mondini dysplasia also showed significant differences at pre-surgery, 1, 3, 6, and 9 months, however, there were no significant differences at 12, 24, and 36 months. Overall, the auditory skills of young children with Mondini dysplasia developed rapidly after cochlear implantation, in a similar manner to that of young children with radiologically normal inner ears. Cochlear implantation is an effective intervention for young children with Mondini dysplasia. [ABSTRACT FROM AUTHOR]

Background: This study presents the characteristics and treatment of small cell lung cancer (SCLC) and an analysis of the factors that impact survival in northeastern Chinese populations, among both smokers and non-smokers. Methods: A retrospective review was performed using 485 Chinese patients diagnosed with pathologically confirmed SCLC diagnoses between January 2001 and December 2007. Data on patient characteristics, treatment patterns, and outcome information was collected systematically. Univariate analysis and the Cox multivariate regression model were used to evaluate prognostic factors. Results: Median survival time was 16 months in all patients, 31 months in limited stage (LS) patients, and 10 months in extensive-stage (ES) patients. Never-smoking patients ( P= 0.0368) with good performance status (PS) ( P= 0.0044) or with normal lactate dehydrogenase (LDH) levels ( P < 0.0001), demonstrated superior survival rates. Multivariate analysis identified that cycles of chemotherapy, PS, LDH levels, recurrence or progression, and clinical stage were each independent prognostic factors applicable to all patients. In LS-SCLC, cycles of chemotherapy were the only prognostic indicator; however, cycles of chemotherapy, LDH levels, and recurrence or progression, were all significant factors in ES-SCLC. Conclusion: Cycles of chemotherapy, PS, LDH levels, recurrence or progression, and clinical stage were proved to be independent prognostic factors for SCLC with variant value based on the SCLC tumor stage. [ABSTRACT FROM AUTHOR]

Conclusion: Most of the infants demonstrated rapid improvement in the three different auditory skills within the first year after switch-on. Infants undergoing hearing aid trial and habilitation demonstrated a significant positive effect on the development of auditory skills in comparison with infants without trial and habilitation. Objective: This paper aims to evaluate the auditory performance of infants of different age at cochlear implantation, emphasize the importance of the hearing aid trial and habilitation before implant, and provide baseline data of auditory development. Methods: In all, 259 infants with prelingually profound hearing loss participated in this study. The Infant-Toddler Meaningful Auditory Integration Scale (IT-MAIS) was used to assess auditory skills in infants at different intervals. Results: The mean scores for the auditory skills improved significantly over time. The mean scores of three skills for each group at each interval were significantly different. The mean scores of the auditory skills were significantly superior for infants undergoing hearing aid trial and habilitation in comparison with those of infants without hearing aid trial and habilitation. [ABSTRACT FROM AUTHOR]

A Pseudomonas aeruginosa (P. aeruginosa) airway infection is one of the predominant causes contributing to the high morbidity and mortality rates in cystic fibrosis (CF) patients. The emergence of antibiotic resistant P. aeruginosa strains has led to an urgent need for new therapeutic approaches. Bacteriophages (phages) are viruses that can infect and lyse specific bacteria, providing a potential alternative approach in targeting antibiotic-resistant strains. We aim to isolate and characterise novel P. aeruginosa phages for combination in a cocktail to kill P. aeruginosa. One particular phage, PA4, could lyse 14/20 clinical isolates as observed through spot assays. This phage could significantly reduce the growth of bacteria in vitro, as determined through planktonic adsorption and inhibition assays as well as crystal violet- and LIVE/DEAD-stained biofilm assays. A morphological and genomic analysis revealed that PA4 belongs to the Myoviridae family and contained 66,450 bp. The broad infectivity profile, good stability in various pH and temperature conditions, lytic ability and the absence of the absences of antibiotic resistance, toxic and lysogenic genes suggest that PA4 is a good candidate for clinical grade use. Overall, phage therapy represents a promising alternative treatment option to antibiotics when treating a P. aeruginosa infection. [ABSTRACT FROM AUTHOR]

Similarly, in nutrient-rich media (BHI, used in laboratory conditions), addition of Tween 80 and oleic acid significantly stimulated I C. accolens i growth (at 0.03125% to 0.5% concentration for Tween 80 and 0.125% to 1% (vol/vol) for oleic acid) compared to untreated control (Fig. Keywords: therapeutics; chronic rhinosinusitis; bacteriology; nasal cavity; Tween 80; oleic acid EN therapeutics chronic rhinosinusitis bacteriology nasal cavity Tween 80 oleic acid 810 813 4 03/25/21 20210401 NES 210401 In health, the human nasal cavity is colonized by a wide variety of commensal bacteria and pathobionts and is dominated by Actinobacteria (mainly I Corynebacteriaceae i ) and Firmicutes (mainly I Staphylococcaceae i ).1,2 In the context of chronic rhinosinusitis (CRS), however, an imbalance or dysbiosis occurs that is characterized by a decreased relative abundance of Actinobacteria and an overgrowth of pathogens such as I Staphylococcus aureus i .3 The fatty acid Tween 80 is commonly used as an excipient in nasal formulations to promote the solubilization of the active drug4 and is approved by the U.S. Food and Drug Administration (FDA) at a maximum concentration of 0.5%.5 It is considered to be well tolerated when delivered to mucosal, intradermal, and intravenous sites.6 Bacteria belonging to the genus Corynebacterium can degrade Tween 80 and use the degradation products (polyoxyethylenic acids and oleic acid) as building blocks to synthesize novel glycolipids that become part of their cell envelope.7 In contrast, fatty acids can also exhibit concentration-dependent anti-microbial activity against pathogens by interfering with cell membrane permeability.8 These unique properties of fatty acid containing formulations and the notion that Tween 80 is already FDA-approved for use in nasal sprays raise the possibility that Tween 80 may have the potential to be used as a prebiotic in topical nasal formulations. [Extracted from the article]

Background: The symptom burden was tremendous and rates of psychological distress were high because of laryngectomy in Laryngeal carcinoma. Anxiety and depression as mainly psychological distress influenced their sleep, pain, and the quality of life (QOL). This study aimed to assess the effiacy of computer-assisted Cognitive Behavioral Therapy (cCBT) for psychological outcomes and QOL in patients with laryngectomy, in addition to overall experience with health care service, during the perioperative period.Methods: A cCBT program was be customized focused on improving anxiety and depressive symptoms among patients with laryngectomy, and then its effectiveness was assessed. Participants were randomly assigned to the TAU (treatment as usual) group (n=40) or CCBT group (cCBT+ TAU, n = 40). The primary outcome measures were the State Anxiety Inventory (SAI), Patients Health Questionnaire-9 (PHQ-9). The secondary outcome measures were the Athens Insomnia Scale (AIS), Visual Analogue Scale-10 (VAS-10). The outcomes were obtained from patients before intervention (T1), 1 hour before surgery (T2), postoperative 3-day (T3), postoperative 7-day (T4), and postoperative 10-day(T5: after intervention completed). Functional Assessment of Cancer Therapy-Head and Neck (FACT-H&N) and satisfaction for health care service were assessed before discharge.Results: A mixed linear model displayed significant improvement in symptoms of anxiety, depression, insomnia, and pain in the two groups (all p<0.001); and revealed a significant decreasing on the SAI, PHQ-9, AIS, and VAS-10 scores in the CCBT group compared to that of TAU group during the post-intervention periods (all p<0.05). Furthermore, the other QOL of patients were higher except for physical well-being (p=0.176) and the satisfaction scores were higher in the CCBT group than that of TAU group (all p<0.05).Conclusion: The new developed cCBT program has a positive effect on psychosomatic symptoms surgery-related among patients with laryngectomy,. And patients with cCBT program reported high levels of QOL and satisfaction during perioperative period. To minimize face-to-face contact, the computer-assisted intervention may be an attractive approach. [ABSTRACT FROM AUTHOR]

Background Childhood cerebral X-linked adrenoleukodystrophy is a rapidly progressive neurodegenerative disorder that affects central nervous system myelin and the adrenal cortex. Hematopoietic stem cell transplantation is the best available curative therapy if performed during the early stages of disease. Only 30% of patients who might benefit from a hematopoietic stem cell transplant will have a full human leukocyte antigen–matched donor, which is considered to be the best choice. Patient Description We present a 5-year-old boy with cerebral X-linked adrenoleukodystrophy whose brain magnetic resonance imaging severity score was 7 and who needed an immediate transplantation without an available full human leukocyte antigen–matched donor. We combined haploidentical and umbilical cord blood sources for transplantation and saw encouraging results. After transplantation, the patient showed neurological stability for 6 months and the level of very long chain fatty acids had decreased. By 1 year, the patient appeared to gradually develop cognition, motor, and visual disturbances resulting from possible mix chimerism. Conclusion Transplantation of haploidentical stem cells combined with the infusion of umbilical cord blood is a novel approach for treating cerebral X-linked adrenoleukodystrophy. It is critical to monitor posttransplant chimerism and carry out antirejection therapy timely for a beneficial clinical outcome. [ABSTRACT FROM AUTHOR]