Effect of deferasirox + erythropoietin vs erythropoietin on erythroid response in Low/Int‐1‐risk MDS patients: Results of the phase II KALLISTO trial

Objectives

Erythropoiesis‐stimulating agents (ESAs) remain first‐choice to treat symptomatic anemia and delay transfusion dependence in most patients with lower‐risk myelodysplastic syndromes (MDS) without del(5q). Deferasirox increased erythroid responses in some lower‐risk MDS patients in clinical trials, and adding low‐dose deferasirox to ESA treatment may further improve erythroid response.

Methods

KALLISTO ( NCT01868477 ) was a randomized, open‐label, multicenter, phase II study. Lower‐risk MDS patients received deferasirox at 10 mg/kg/d (dispersible tablets) or 7 mg/kg/d (film‐coated tablets) plus erythropoietin (n = 11), or erythropoietin alone (n = 12) for 24 weeks. The primary endpoint was the between‐group difference in erythroid response within 12 weeks.

Results

Erythroid response occurred in 27.3% of patients receiving deferasirox plus erythropoietin vs 41.7% of patients receiving erythropoietin alone within 12 weeks (difference 14.4%; 95% CI −24.0, 48.16). Within 24 weeks, the hematologic response rate was 27.3% with deferasirox plus erythropoietin vs 50% with erythropoietin alone, and hematologic improvement rates were 45.5% vs 100%. Deferasirox plus erythropoietin was generally well tolerated.

Conclusions

In this small pilot study, combining low‐dose deferasirox with erythropoietin did not improve erythroid response. It remains of interest to investigate early chelation approaches with even lower deferasirox doses plus erythropoietin in lower‐risk MDS patients before the onset of transfusion dependence.

     

Characteristics of MDS patients seen at private practices differ significantly from those treated at university hospitals in Germany

Background

Myelodysplastic syndromes (MDS) are mainly a disease of the elderly. Commonly, MDS patients are treated in an outpatient setting making hematological/oncological private practices (PP) an important backbone in the management of MDS patients.

Methods

To gain more insights into the characteristics of patients with MDS treated in hematological/oncological PP and to evaluate the daily diagnostic routines and classification systems used, we performed questionnaire-based analyses. Moreover, to investigate whether characteristics of MDS in PP differ from patients treated in specialized MDS centers in university hospitals (UH), we compared both cohorts of MDS patients.

Results

In total, 197 patients in PP and 165 patients in UH were enrolled. Patients in UH were significantly younger as compared to PP. Furthermore, in UH, a greater proportion of patients with international prognostic scoring system (IPSS) higher risk were found, whereas patients with IPSS lower risk were more frequent in PP. In addition, patients in UH had significantly lower hemoglobin levels and platelet counts compared to PP.

Conclusion

Our data show that PP and UH are approached by different MDS patient cohorts resulting in different diagnostic workups of MDS patients.     

Therapy with low‐dose azacitidine for MDS in children and young adults: a retrospective analysis of the EWOG‐MDS study group

Low‐dose azacitidine is efficient and safe in the therapy of malignant myeloid disorders in adults but data in children are lacking. We present a retrospective analysis of 24 children and young adults with myelodysplastic syndrome (MDS) who received azacitidine at the time of first diagnosis or relapse after allotransplant (2 children were treated with azacitidine both initially and for relapse). Diagnoses were refractory cytopenia of childhood (N = 4), advanced primary MDS (N = 9) and secondary MDS (N = 11). The median duration of treatment was four cycles. Azacitidine was well tolerated, but cytopenias led to dose reduction in five cases. Treatment was discontinued in one child because of impaired renal function. Sixteen MDS patients were treated with azacitidine at first diagnosis. One complete clinical remission was observed and one child showed complete marrow remission; six children experienced stable disease with haematological improvement. Ten children received azacitidine for relapsed MDS after transplant: of these, seven experienced stable disease for 2–30 cycles (median 3), including one patient with haematological improvement for seven cycles. In summary, azacitidine is effective in some children with MDS and appears to be a non‐toxic option in palliative situations to prolong survival.     

Prognostic scoring systems for myelodysplastic syndromes (MDS) in a population‐based setting: a report from the Swedish MDS register

The myelodysplastic syndromes (MDS) have highly variable outcomes and prognostic scoring systems are important tools for risk assessment and to guide therapeutic decisions. However, few population‐based studies have compared the value of the different scoring systems. With data from the nationwide Swedish population‐based MDS register we validated the International Prognostic Scoring System (IPSS), revised IPSS (IPSS‐R) and the World Health Organization (WHO) Classification‐based Prognostic Scoring System (WPSS). We also present population‐based data on incidence, clinical characteristics including detailed cytogenetics and outcome from the register. The study encompassed 1329 patients reported to the register between 2009 and 2013, 14% of these had therapy‐related MDS (t‐MDS). Based on the MDS register, the yearly crude incidence of MDS in Sweden was 2·9 per 100 000 inhabitants. IPSS‐R had a significantly better prognostic power than IPSS (P < 0·001). There was a trend for better prognostic power of IPSS‐R compared to WPSS (P = 0·05) and for WPSS compared to IPSS (P = 0·07). IPSS‐R was superior to both IPSS and WPSS for patients aged ≤70 years. Patients with t‐MDS had a worse outcome compared to de novo MDS (d‐MDS), however, the validity of the prognostic scoring systems was comparable for d‐MDS and t‐MDS. In conclusion, population‐based studies are important to validate prognostic scores in a ‘real‐world’ setting. In our nationwide cohort, the IPSS‐R showed the best predictive power.     

Mammalian‐target of rapamycin inhibition with temsirolimus in myelodysplastic syndromes (MDS) patients is associated with considerable toxicity: results of the temsirolimus pilot trial by the German MDS Study Group (D‐MDS)

The mammalian‐target of rapamycin (also termed mechanistic target of rapamycin, mTOR) pathway integrates various pro‐proliferative and anti‐apoptotic stimuli and is involved in regulatory T‐cell (TREG) development. As these processes contribute to the pathogenesis of myelodysplastic syndromes (MDS), we hypothesized that mTOR modulation with temsirolimus (TEM) might show activity in MDS. This prospective multicentre trial enrolled lower and higher risk MDS patients, provided that they were transfusion‐dependent/neutropenic or relapsed/refractory to 5‐azacitidine, respectively. All patients received TEM at a weekly dose of 25 mg. Of the 9 lower‐ and 11 higher‐risk patients included, only 4 (20%) reached the response assessment after 4 months of treatment and showed stable disease without haematological improvement. The remaining patients discontinued TEM prematurely due to adverse events. Median overall survival (OS) was not reached in the lower‐risk group and 296 days in the higher‐risk group. We observed a significant decline of bone marrow (BM) vascularisation (P = 0·006) but were unable to demonstrate a significant impact of TEM on the balance between TREG and pro‐inflammatory T‐helper‐cell subsets within the peripheral blood or BM. We conclude that mTOR‐modulation with TEM at a dose of 25 mg per week is accompanied by considerable toxicity and has no beneficial effects in elderly MDS patients.     

Tumor suppressor gene BLU is frequently downregulated by promoter hypermethylation in myelodysplastic syndrome

Purpose  

BLU methylation status was investigated in bone marrow mononuclear cells from newly diagnosed myelodysplastic syndrome (MDS) patients and patients who received 5-aza-2′-deoxycytidine (decitabine) treatment so as to determine the effect of BLU in the pathogenesis of MDS.     

骨髓增生异常综合征患者骨髓不同细胞亚群凋亡的研究

目的:观察骨髓增生异常综合征(MDS)患者骨髓单个核细胞(BMNC)及不同细胞亚群凋亡情况。方法:应用流式细胞仪、CD45单抗、Annexin-V/PI检测15例MDS患者骨髓细胞凋亡情况。结果:MDS患者BMNC凋亡(5.57％)明显高于对照组(3.19％)(P<0.05),淋巴细胞(R_1)凋亡(20.82％)明显高于对照组(9.98％)(P<0.05),且各细胞亚群之间比较,淋巴细胞(R_1)凋亡明显高于原始/粒细胞群(R_2)(10.22％)和红细胞群(R_3)(6.66％),差异有显著性意义(P<0.05)。红系凋亡并不增加,与原始/粒细胞群比较,差异无显著性意义(P>0.05)。结论:MDS患者骨髓淋巴细胞凋亡增加,且可能为CD4~+T细胞(Th),从而使T细胞亚群Th/Ts比例失调,造成免疫调节异常,提示这种异常在MDS发病过程中起重要作用。     

Comparing the Functional Independence Measure and the interRAI/MDS for use in the functional assessment of older adults: a review of the literature

Background

The rehabilitation of older persons is often complicated by increased frailty and medical complexity - these in turn present challenges for the development of health information systems. Objective investigation and comparison of the effectiveness of geriatric rehabilitation services requires information systems that are comprehensive, reliable, valid, and sensitive to clinically relevant changes in older persons. The Functional Independence Measure is widely used in rehabilitation settings - in Canada this is used as the central component of the National Rehabilitation Reporting System of the Canadian Institute of Health Information. An alternative system has been developed by the interRAI consortium. We conducted a literature review to compare the development and measurement properties of these two systems.

Methods

English language literature published between 1983 (initial development of the FIM) and 2008 was searched using Medline and CINAHL databases, and the reference lists of retrieved articles. Relevant articles were summarized and charted using the criteria proposed by Streiner. Additionally, attention was paid to the ability of the two systems to address issues particularly relevant to older rehabilitation clients, such as medical complexity, comorbidity, and responsiveness to small but clinically meaningful improvements.

Results

In total, 66 articles were found that met the inclusion criteria. The majority of FIM articles studied inpatient rehabilitation settings; while the majority of interRAI/MDS articles focused on nursing home settings. There is evidence supporting the reliability of both instruments. There were few articles that investigated the construct validity of the interRAI/MDS.

Conclusion

Additional psychometric research is needed on both the FIM and MDS, especially with regard to their use in different settings and with different client groups.     

Phase II study of tosedostat with cytarabine or decitabine in newly diagnosed older patients with acute myeloid leukaemia or high‐risk MDS

Tosedostat, an oral aminopeptidase inhibitor, has synergy with cytarabine and hypomethylating agents. We performed a Phase II trial to determine rates of complete remission (CR) and survival using tosedostat with cytarabine or decitabine in older patients with untreated acute myeloid leukaemia (AML) or high‐risk myelodysplastic syndrome (MDS). Thirty‐four patients ≥60 years old (median age 70 years; range, 60–83) were randomized to receive tosedostat (120 mg on days 1–21 or 180 mg continuously) with 5 d of either cytarabine (1 g/m²/d) or decitabine (20 mg/m²/d) every 35 d. Twenty‐nine patients (85%) had AML, including 15 (44%) with secondary AML/MDS, and 5 (15%) had MDS‐refractory anaemia with excess blasts type 2. The CR/CR with incomplete count recovery (CRi) rate was 53% [9 in each arm; 14 CR (41%) and 4 CRi (12%)], attained in 6 of 14 patients with adverse cytogenetics and 4 of 7 with FLT3‐internal tandem duplication mutations. Median follow‐up was 11·2 months (range, 0·5–22·3), and median survival was 11·5 months (95% confidence interval, 5·2–16·7). Twenty‐three patients (67·6%) were treated as outpatients and 10 of these patients required hospitalization for febrile neutropenia. No Grade 3–4 non‐haematological toxicities required withdrawal from study. Tosedostat with cytarabine or decitabine is tolerated in older patients with untreated AML/MDS, results in a CR/CRi rate of >50%, and warrants further study in larger trials.     

Telomere length is an independent prognostic marker in MDS but not in de novo AML

Telomere dysfunction is implicated in the generation of large‐scale genomic rearrangements that drive progression to malignancy. In this study we used high‐resolution single telomere length analysis (STELA) to examine the potential role of telomere dysfunction in 80 myelodysplastic syndrome (MDS) and 95 de novo acute myeloid leukaemia (AML) patients. Despite the MDS cohort being older, they had significantly longer telomeres than the AML cohort (P < 0·0001) where telomere length was also significantly shorter in younger AML patients (age <60 years) (P = 0·02) and in FLT3 internal tandem duplication‐mutated AML patients (P = 0·03). Using a previously determined telomere length threshold for telomere dysfunction (3·81 kb) did not provide prognostic resolution in AML [Hazard ratio (HR) = 0·68, P = 0·2]. In contrast, the same length threshold was highly prognostic for overall survival in the MDS cohort (HR = 5·0, P < 0·0001). Furthermore, this telomere length threshold was an independent parameter in multivariate analysis when adjusted for age, gender, cytogenetic risk group, number of cytopenias and International Prognostic Scoring System (IPSS) score (HR = 2·27, P < 0·0001). Therefore, telomere length should be assessed in a larger prospective study to confirm its prognostic role in MDS with a view to integrating this variable into a revised IPSS.     

Surgical treatment for esophageal cancer patients with myelodysplastic syndrome

Background

Myelodysplastic syndrome (MDS) is a clinical condition with pancytopenia, dysfunction of neutrophils and poor prognosis caused by dysplasia of the bone marrow. MDS patients tend to have other malignant diseases, and the treatment is complicated because of high morbidity and mortality. Moreover, esophageal cancer is one of the most aggressive cancers, and its surgical treatment has high morbidity.

Methods

Among 450 patients with esophageal cancer who underwent surgical treatment, 4 (0.8 %) had MDS. We describe esophageal cancer patients with MDS who underwent radical surgical treatment and estimate the perioperative management and postoperative outcome.

Results

Two patients underwent transhiatal resection involving lower esophagectomy and proximal gastrectomy, and two other patients underwent thoracoscopic thoracoabdominal resection. Important critical points for the surgical treatment of esophageal cancer patients with MDS were as follows: hematological examination for patients with cytopenia, surgical indication for patients with WHO classification of RA or RARS and IPSS of low or intermediate-1 risk, planning transfusions for patients with thrombocytopenia, expecting postoperative complications such as pneumonia or bleeding, and careful follow-up for the early detection of relapse of disease.

Conclusion

With careful management, we were able to treat esophageal cancer patients with MDS surgically.     

The prognostic impact of variant allele frequency (VAF) in TP53 mutant patients with MDS: A systematic review and meta-analysis

Tumor protein p53 (TP53) is frequently expressed in patients with myelodysplastic syndromes (MDS). Studies have already reported the poor prognostic impact of TP53 gene mutations in MDS patients. However, parts of this subgroup of patients with low-risk MDS still have relatively better survival and longer remission times. Therefore, we performed a meta-analysis to evaluate the prognostic difference intra-gene of variant allele frequency (VAF). The primary endpoint was overall survival (OS), and event-free survival (EFS) was selected as the secondary endpoint. We extracted the hazard ratio (HR) and 95% confidence interval (CI) for OS and EFS from univariate and multivariate Cox proportional hazard models. A total of 4003 MDS patients and 1278 TP53-mutated patients from 13 cohorts of 11 studies up to February 24, 2020, were included in our meta-analysis. Pooled HRs suggested that a high mutant VAF had an adverse impact on OS (HR = 2.11, 95% CI: 1.48-3.01, P < .0001) but no impact on EFS (HR = 15.57, 95% CI: 0.75-324.44, P = .003) in MDS patients. Twenty percent is a proper threshold to set (HR = 2.02, 95% CI: 1.31-3.13, P = .001) and is a rough line between high clone burden and low clone burden, while 40% is an exact cutoff point (HR = 2.11, 95% CI: 1.26-3.55, P < .0001) to guide diagnosis and treatment. Beyond the traditional binary classification of gene mutation, we aimed to find a way to divide mutant molecular markers more specifically by VAF to provide clinical therapeutic values. Our meta-analysis indicates that a high VAF is an independent, adverse prognostic factor for OS in TP53 mutant MDS patients. Patients with mediate/low-frequency parts who could be treated like wide-type patients have relatively better survival and may choose allogeneic hematopoietic stem cell transplantation as conditions permitting. Further prospective studies are needed in the future, and a large subgroup analysis of the same cutoff point subgroups is needed to obtain a more reliable basis for the impact of other mutant gene VAFs on the prognosis of MDS.     

Coomb’s阳性的MDS病例报道及MDS的诊断思考

MDS是血液科的常见疾病,疾病的本质是①髓系的发育异常,即无效造血,临床表现为一系或多系的血细胞减少;②高危演变为急性髓系白血病,在骨髓象表现为原始细胞增多。并且还需要排除其他可能引起血细胞减少的疾病,这使得临床上诊断有一定困难。随着对疾病本质的深入理解及新分子诊断技术的应用,可以寻找更多MDS的证据,目前对MDS的诊断水平已经大大提高。但是在临床上仍有一些起初表现不典型,诊断十分困难的MDS,为早期治疗造成困难,现报道我们临床上收治的以Coomb’s阳性的自身免疫性溶血为早期表现,疾病进展迅速的MDS患者,以加强我们对MDS的疾病认识。     

Azacitidine improves outcome in higher‐risk MDS patients with chromosome 7 abnormalities: a retrospective comparison of GESMD and GFM registries

Treatment with azacitidine (AZA) has been suggested to be of benefit for higher‐risk myelodysplastic syndrome (HR‐MDS) patients with chromosome 7 abnormalities (Abn 7). This retrospective study of 235 HR‐MDS patients with Abn 7 treated with AZA (n = 115) versus best supportive care (BSC; n = 120), assessed AZA treatment as a time‐varying variable in multivariable analysis. A Cox Regression model with time‐interaction terms of overall survival (OS) at different time points confirmed that, while chromosome 7 cytogenetic categories (complex karyotype [CK] versus non‐CK) and International Prognostic Scoring System risk (high versus intermediate‐2) retained poor prognosis over time, AZA treatment had a favourable impact on OS during the first 3 years of treatment compared to BSC (Hazard ratio [HR] 0·5 P < 0·001 at 1 year, 0·7 P = 0·019 at 2 years; 0·73 P = 0·029 at 3 years). This benefit was present in all chromosome 7 categories, but tended to be greater in patients with CK (risk reduction of 82%, 68% and 53% at 1, 3 and 6 months in CK patients; 79% at 1 month in non‐CK patients, P < 0·05 for all). AZA also significantly improved progression‐free survival (P < 0·01). This study confirms a time‐dependent benefit of AZA on outcome in patients with HR‐MDS and cytogenetic abnormalities involving chromosome 7, especially for those with CK.     

Hypocellular myelodysplastic syndromes (MDS): new proposals

Summary. To determine whether hypocellular MDS differs from normo/hypercellular MDS, we attempted to identify hypocellular MDS cases either by correcting the bone marrow (BM) cellularity by age (28 patients) or by using a single arbitrary value of BM cellularity (25 patients) and compared these two groups of hypocellular cases to the normo/hypercellular MDS cases (72 patients). 18 patients were common to both hypocellular groups. Patients with hypocellular MDS in both of these selected groups have similar features with regard to age and sex distribution, peripheral blood and bone marrow parameters, FAB subtypes, karyotypes, leukaemic transformation, and survival. However, the median age of patients in < 30% BM cellularity group was higher than those patients in the age-corrected group (69 years v 62 years). The selection of < 30% cellularity excluded 10 cases in the age group < 70 years but included another seven patients in the age group of > 70 years. However, correction of BM cellularity by age revealed that those included patients (selected for < 30% cellularity) who had normocellular BM by their age. Therefore we recommend the age-correcting grouping to ensure comparable series for comparison, for response to treatment, and survival. Finally, BM cellularity does not appear to be an important factor on prognosis in MDS, because patients with hypocellular MDS in both selected groups have similar prognosis to those with normo/hypercellular MDS patients.     

Hospitalization and emergency department visits among seniors receiving homecare: a pilot study

Background

The objective of this study was to examine the Minimum Data Set (MDS) and Geriatric Depression Scale (GDS) as measures of depression among nursing home residents.

Methods

The data for this study were baseline, pre-intervention assessment data from a research study involving nine nursing homes and 704 residents in Massachusetts. Trained research nurses assessed residents using the MDS and the GDS 15-item version. Demographic, psychiatric, and cognitive data were obtained using the MDS. Level of depression was operationalized as: (1) a sum of the MDS Depression items; (2) the MDS Depression Rating Scale; (3) the 15-item GDS; and (4) the five-item GDS. We compared missing data, floor effects, means, internal consistency reliability, scale score correlation, and ability to identify residents with conspicuous depression (chart diagnosis or use of antidepressant) across cognitive impairment strata.

Results

The GDS and MDS Depression scales were uncorrelated. Nevertheless, both MDS and GDS measures demonstrated adequate internal consistency reliability. The MDS suggested greater depression among those with cognitive impairment, whereas the GDS suggested a more severe depression among those with better cognitive functioning. The GDS was limited by missing data; the DRS by a larger floor effect. The DRS was more strongly correlated with conspicuous depression, but only among those with cognitive impairment.

Conclusions

The MDS Depression items and GDS identify different elements of depression. This may be due to differences in the manifest symptom content and/or the self-report nature of the GDS versus the observer-rated MDS. Our findings suggest that the GDS and the MDS are not interchangeable measures of depression.     

Deferasirox chelation therapy in patients with transfusion‐dependent MDS: a ‘real‐world’ report from two regional Italian registries: Gruppo Romano Mielodisplasie and Registro Basilicata

Deferasirox (DFX) is an orally administered iron chelator approved for use in patients with transfusion‐dependent iron overload due to myelodysplastic syndromes (MDS). The safety and efficacy of DFX has been explored in clinical trial settings, but there is little data on unselected patients with MDS. The aim of this study was to retrospectively evaluate the safety, compliance, efficacy and effect on haematopoiesis of DFX in a large ‘real‐world’ MDS population. One hundred and eighteen patients with transfusion‐dependent MDS were treated with DFX across 11 centres in Italy. Serum ferritin levels, haematological response, dosing, adverse events and transfusion dependence were recorded at baseline, 3, 6, 12 and 24 months following initiation of treatment. DFX reduced mean serum ferritin levels from 1790 to 1140 ng/mL (P < 0.001), with 7.1% of patients achieving transfusion independence. Significant haematological improvement was seen in erythroid (17.6%), platelet (5.9%) and neutrophil counts (7.1%). Adverse events were reported in 47.5% of patients, including gastrointestinal and renal toxicity. Regression analysis showed that higher starting doses of DFX are associated with transfusion independence at 24 months. DFX is a safe, effective treatment for transfusion‐dependent MDS that can lead to transfusion independence and haematological improvement in a subset of patients.     

Prospective analysis of clinical and cytogenetic features of 435 cases of MDS diagnosed using the WHO (2001) classification: a prognostic scoring system for predicting survival in RCMD

We characterized the prevalence, clinical and cytogenetic characteristics and survival of 435 patients diagnosed with de novo MDS in a single laboratory according to WHO criteria, and compared the utility of different scoring systems to predict survival for individual subtypes of MDS. The mean follow-up period was 25.1 (5.5–53.2) months. Our results confirm major differences in the age-distribution and prevalence of individual subtypes of MDS between Asian and Western patients with a median age of 58 years and a predominance of RCMD (69.9%). Survival rates were similar to those reported in the West: the 3-year survival rate for MDS was 46.7% with a median survival time for RCMD of 38 months and RAEB, 10 months. We found that the IPSS and WPSS scoring systems, which are weighted heavily by blast cell count and karyotype, were not independent predictors for survival in RCMD patients. Multivariate analysis demonstrated that a scoring system based on age (≥60 years), ANC (<1.0 × 10⁹/L), Hb (<90 g/L), number of cytopenias and complex karyotype is a more useful predictor of survival in RCMD.     

Mean corpuscular volume predicts prognosis in MDS patients with abnormal karyotypes

The prognostic value of karyotype in patients with myelodysplastic syndrome (MDS) is generally appreciated. However, the factors that are predictive of prognosis of patients with abnormal karyotypes are not known. In this study, we evaluated the prognostic value of International Prognostic Scoring System (IPSS) and World Health Organization classification-based prognostic scoring system (WPSS) in 164 adult MDS patients with abnormal karyotypes. We also analyzed the prognostic relevance of mean corpuscular volume (MCV) in these patients. We found that both IPSS and WPSS had strong prognostic value in patients with abnormal karyotypes (P < 0.001, P < 0.001). Furthermore, we observed the significant differences in the survival of patients with abnormal karyotypes based on MCV stratification: The median survival of patients with macrocytosis was 31.0 months, significantly longer than the 16.5-month median survival time of patients with MCVs of less than 100 fl (P = 0.001). Multivariate analysis revealed that lower level of hemoglobin (P = 0.012, HR = 6.83), higher level of marrow blasts (P < 0.001, HR = 1.93), complex karyotype (P = 0.001, HR = 3.32), and MCV of less than 100 fl (P = 0.026, HR = 1.75) were independent risk factors that affected the survival of patients with abnormal karyotypes.     

Evaluating clinical stop‐smoking services globally: towards a minimum data set

Background and aims

Behavioural and pharmacological support for smoking cessation improves the chances of success and represents a highly cost‐effective way of preventing chronic disease and premature death. There is a large number of clinical stop‐smoking services throughout the world. These could be connected into a global network to provide data to assess what treatment components are most effective, for what populations and in what settings. To enable this, a minimum data set (MDS) is required to standardize the data captured from smoking cessation services globally.

Methods

We describe some of the key steps involved in developing a global MDS for smoking cessation services and methodologies to be considered for their implementation, including approaches for reaching consensus on data items to include in a MDS and for its robust validation. We use informal approximations of these methods to produce an example global MDS for smoking cessation. Our aim with this is to stimulate further discussion around the development of a global MDS for smoking cessation services.

Results

Our example MDS comprises three sections. The first is a set of data items characterizing treatments offered by a service. The second is a small core set of data items describing clients’ characteristics, engagement with the service and outcomes. The third is an extended set of client data items to be captured in addition to the core data items wherever resources permit.

Conclusions

There would be benefit in establishing a minimum data set (MDS) to standardize data captured for smoking cessation services globally. Once implemented, a formal MDS could provide a basis for meaningful evaluations of different smoking cessation treatments in different populations in a variety of settings across many countries.